Stem Cell Biology Overview

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Questions and Answers

What is a consequence of a chromosomal nondisjunction event during gametogenesis?

  • It results in the normal number of chromosomes.
  • It occurs only in somatic cells.
  • It can lead to the presence of an extra chromosome. (correct)
  • It prevents the formation of fertilized eggs.

Which trisomy is characterized by three copies of chromosome 18?

  • Down Syndrome
  • Patau Syndrome
  • Turner Syndrome
  • Edwards Syndrome (correct)

What percentage of known pregnancies are estimated to end in miscarriage?

  • 30 to 40 percent
  • 10 to 20 percent (correct)
  • 70 to 80 percent
  • 50 to 60 percent

What is the primary risk factor for trisomy 13 and trisomy 18 during reproduction?

<p>Advanced maternal age (C)</p> Signup and view all the answers

What is the typical fate of babies born with trisomy 13?

<p>Most do not survive beyond their teens. (C)</p> Signup and view all the answers

What percentage of fertilized eggs are estimated to be lost before or during implantation?

<p>30 to 50 percent (B)</p> Signup and view all the answers

Which statement about embryonic stem cells is accurate?

<p>They are derived from nonviable embryos. (A)</p> Signup and view all the answers

What does the recent ruling by Alabama's supreme court assert about embryos?

<p>They are classified as 'extrauterine children'. (A)</p> Signup and view all the answers

What is the approximate likelihood of long-term disease-free survival for patients with acute myeloid leukemia who receive BMT in first remission?

<p>50 to 60% (A)</p> Signup and view all the answers

What is the primary role of the Encaptra System?

<p>To protect PEC-01 cells from the host immune system. (D)</p> Signup and view all the answers

Which condition is NOT typically treated with BMSC transplantation?

<p>Hypertension (C)</p> Signup and view all the answers

What is the probability of relapse after a BMT for a patient with acute leukemia who is in first remission?

<p>20% (A)</p> Signup and view all the answers

What material is the Encaptra System membrane made of?

<p>Expanded PTFE composite (D)</p> Signup and view all the answers

For successful BMT, the donor and recipient must have nearly identical variants of which complex?

<p>Human Leukocyte Antigen (HLA) (D)</p> Signup and view all the answers

What challenge does fibrosis pose to the Encaptra System?

<p>It limits diffusion of insulin and other hormones. (A)</p> Signup and view all the answers

How many known alleles of HLA-D are there?

<p>563 (A)</p> Signup and view all the answers

How does the membrane of the Encaptra System function?

<p>It selectively allows oxygen and nutrients in while letting insulin out. (D)</p> Signup and view all the answers

What is the estimated occurrence rate of finding a matching donor for BMT?

<p>1 in 100,000 (B)</p> Signup and view all the answers

What is the primary goal of the collaboration with Gore concerning the Encaptra System?

<p>To engineer materials that promote vascularization. (D)</p> Signup and view all the answers

What happens when enough collagenous material surrounds the Encaptra System?

<p>It greatly limits diffusion of substances in and out. (D)</p> Signup and view all the answers

What is the key limiting factor in the use of BMT?

<p>Lack of donors (A)</p> Signup and view all the answers

What condition could the stem cell transformation technique from the Stice lab potentially benefit?

<p>Neural injuries from trauma. (A)</p> Signup and view all the answers

What percentage of long-term survival is achievable for chronic myelocytic leukemia patients who receive BMT during the remission phase?

<p>60 to 70% (C)</p> Signup and view all the answers

What is fibrosis primarily characterized by?

<p>Excess fibrous connective tissue formation. (C)</p> Signup and view all the answers

What role does phosphorylation play in glucose uptake by beta cells?

<p>It prevents glucose from diffusing back across the cell membrane. (A)</p> Signup and view all the answers

What is the significance of the Km value for GLUT2 in glucose transport?

<p>It signifies rapid glucose transport above a specific blood concentration. (C)</p> Signup and view all the answers

What triggers the opening of voltage-gated calcium ion channels in beta cells?

<p>An increase in blood glucose concentration past a specific threshold. (B)</p> Signup and view all the answers

What occurs at lower blood sugar levels in the alpha cell?

<p>Glycolysis initiation (D)</p> Signup and view all the answers

What is a potential benefit of using ESC-derived treatment in diabetes?

<p>They can respond to changes in blood sugar levels effectively. (D)</p> Signup and view all the answers

What happens when the threshold of 5.5 nmol/l of glucose is exceeded?

<p>Insulin loaded vesicles are triggered to release insulin. (D)</p> Signup and view all the answers

Which of the following accurately describes GLUT2's function?

<p>It facilitates rapid glucose transport under specific conditions. (A)</p> Signup and view all the answers

What is a common characteristic of tissue uptake of glucose?

<p>It can occur through facilitated diffusion. (B)</p> Signup and view all the answers

What components comprise the glucose sensor of the alpha cell?

<p>GLUT1 and glucokinase (B)</p> Signup and view all the answers

What is the Km value for glucokinase in alpha cells?

<p>5.5 mM (D)</p> Signup and view all the answers

How do stem-cell-derived beta cells (SC-β) aid in diabetes treatment?

<p>They flux calcium ions in reaction to glucose and secrete insulin. (B)</p> Signup and view all the answers

Which genetic condition is linked to alterations in the GLUT1 transporter?

<p>GLUT1 deficiency syndrome (C)</p> Signup and view all the answers

What is the primary source of carbon and energy for the brain?

<p>Glucose (A)</p> Signup and view all the answers

Which of the following is a symptom of common GLUT1 deficiency syndrome?

<p>Frequent seizures (D)</p> Signup and view all the answers

What may be the first sign of GLUT1 deficiency syndrome in newborns?

<p>Involuntary eye movements (C)</p> Signup and view all the answers

What condition can result from GLUT1 deficiency syndrome?

<p>Microcephaly (B)</p> Signup and view all the answers

What characterizes type 1 diabetes?

<p>The pancreas produces little or no insulin. (C)</p> Signup and view all the answers

Which of the following symptoms are commonly associated with high blood sugar levels?

<p>Increased thirst and hunger (D)</p> Signup and view all the answers

What condition can type 2 diabetes lead to over time?

<p>Serious damage to various organs (D)</p> Signup and view all the answers

What is a common neurological problem associated with diabetes?

<p>Spasticity (A)</p> Signup and view all the answers

What does the body lack in type 1 diabetes that prevents sugar absorption?

<p>Insulin (B)</p> Signup and view all the answers

Which factor contributes to the rising prevalence of type 2 diabetes?

<p>Aging population (D)</p> Signup and view all the answers

What is the main factor that leads to confusion or lethargy in some diabetes patients?

<p>Low sugar levels during fasting (D)</p> Signup and view all the answers

How is type 2 diabetes often categorized?

<p>Non-insulin-dependent diabetes (D)</p> Signup and view all the answers

Which type of stem cells can develop into a complete organism?

<p>Totipotent (D)</p> Signup and view all the answers

Pluripotent stem cells can only differentiate into a limited number of cell types.

<p>False (B)</p> Signup and view all the answers

What term describes stem cells that have yet to specialize and have no specific function?

<p>unspecialized</p> Signup and view all the answers

________ stem cells are derived from fetal tissue, cord blood, and adult stem cells.

<p>Multipotent</p> Signup and view all the answers

Match the type of stem cells with their descriptions:

<p>Totipotent = Can develop into a new individual Pluripotent = Can form any cell type in the body Multipotent = Limited to differentiating into a few related cell types</p> Signup and view all the answers

What is one of the sources of embryonic stem cells?

<p>Inner cell mass of the blastocyst (C)</p> Signup and view all the answers

Induced pluripotent stem cells are derived from adult stem cells.

<p>False (B)</p> Signup and view all the answers

Stem cells can be classified based on their ability to ________.

<p>differentiate</p> Signup and view all the answers

What type of cells are formed from the inner cell mass of an embryo?

<p>Embryonic stem cells (A)</p> Signup and view all the answers

Non-viable embryos are typically used to create embryonic stem cell cultures.

<p>True (A)</p> Signup and view all the answers

What is the purpose of coaxing embryonic stem cells?

<p>To form differentiated cell types for potential medical uses.</p> Signup and view all the answers

Embryonic cell lines are cultured under ______ conditions to promote differentiation.

<p>special</p> Signup and view all the answers

Match the following types of embryos with their description:

<p>Single-celled embryo = B Embryo with a central cavity = G Embryo showing degeneration = A Embryo with multiple small blastomeres = E</p> Signup and view all the answers

What happens to unused embryos created during IVF?

<p>They are either destroyed or donated for medical use. (C)</p> Signup and view all the answers

Most human embryos produced naturally are viable.

<p>False (B)</p> Signup and view all the answers

What chromosomal abnormality is indicated by blue dots in the cell samples?

<p>Trisomy.</p> Signup and view all the answers

What is the outcome of a chromosomal nondisjunction event?

<p>It results in a gamete with an extra chromosome. (A)</p> Signup and view all the answers

Trisomy 18 (Edwards Syndrome) is a condition where most babies die before they are born.

<p>True (A)</p> Signup and view all the answers

What percentage of pregnancies are estimated to reach full term?

<p>30%</p> Signup and view all the answers

A chromosomal trisomy involves having three copies of a __________ instead of the normal two.

<p>chromosome</p> Signup and view all the answers

Match the following trisomy conditions with their outcomes:

<p>Trisomy 13 = Rarely lives into their teens Trisomy 18 = Majority die within 5 to 15 days Down Syndrome = Can live into adulthood Trisomy 21 = Most common form of trisomy</p> Signup and view all the answers

What factor contributes to the loss of fertilized eggs before implantation?

<p>Chromosomal abnormalities (A)</p> Signup and view all the answers

A single cell can be removed from a 2-3 day old embryo without affecting its viability.

<p>True (A)</p> Signup and view all the answers

What is the legal classification of embryos as ruled by Alabama's supreme court?

<p>Extrauterine children</p> Signup and view all the answers

Which of the following conditions can be treated with BMSC transplantation?

<p>Acute myeloid leukemia (D)</p> Signup and view all the answers

Patients with sickle cell disease are not candidates for BMT.

<p>False (B)</p> Signup and view all the answers

What is the approximate likelihood of long-term disease-free survival for patients with acute lymphoblastic leukemia who receive BMT in first remission?

<p>approximately 50 to 60%</p> Signup and view all the answers

To minimize rejection, the donor and recipient should share nearly identical gene variants for the __________ complex.

<p>Human Leukocyte Antigen</p> Signup and view all the answers

What is the probability of relapse for patients with acute leukemia who are in first remission at the time of transplant?

<p>20% (C)</p> Signup and view all the answers

Pediatric BMT has grown in popularity due to its potential for curing genetic diseases.

<p>True (A)</p> Signup and view all the answers

Match the following diseases with their treatment method:

<p>Sickle Cell Disease = BMSC transplantation Chronic myelocytic leukemia = BMT Thalassemia = Bone marrow transplant Aplastic anemia = Stem cell therapy</p> Signup and view all the answers

What is the likelihood of finding a matching donor for BMT?

<p>1 in 100,000</p> Signup and view all the answers

What is the probability of finding a match for ethnic minorities in the NMDP registry?

<p>30-40% (B)</p> Signup and view all the answers

Caucasians currently have an 80% chance of finding a match in the donor registry.

<p>True (A)</p> Signup and view all the answers

What is the role of calcium ions in insulin release?

<p>Calcium ions trigger the fusion of insulin vesicles with the plasma membrane for release.</p> Signup and view all the answers

The odds of finding a random HLA match are approximately 1 in __________.

<p>100,000</p> Signup and view all the answers

What substance is critical for the treatment of diabetes by being released into the blood?

<p>Insulin (D)</p> Signup and view all the answers

Match the following terms with their definitions:

<p>Hematopoietic stem cells = Develop into blood cells Inducible calcium ion channel = Triggers calcium influx Insulin granules = Vesicles that contain insulin Mechanosensitive ion channels = Activated by mechanical stimuli</p> Signup and view all the answers

What percentage of registered donors are South Asians?

<p>2% (D)</p> Signup and view all the answers

Mechanical stimuli can activate calcium influx through ion channels.

<p>True (A)</p> Signup and view all the answers

What is the purpose of the Encaptra System?

<p>To protect PEC-01 cells from the host immune system (A)</p> Signup and view all the answers

The Encaptra System uses a solid barrier that prevents the diffusion of oxygen and nutrients.

<p>False (B)</p> Signup and view all the answers

What is the primary material used in the construction of the Encaptra System's membrane?

<p>Expanded PTFE composite</p> Signup and view all the answers

Fibrosis is the formation of excess _____ connective tissue in an organ or tissue.

<p>fibrous</p> Signup and view all the answers

Match the following components of the Encaptra System with their functions:

<p>PEC-01 cells = Insulin production Semi-permeable membrane = Allows diffusion of nutrients Scar tissue = Limits diffusion Vascular network = Facilitates blood flow</p> Signup and view all the answers

What challenge does fibrosis pose for the Encaptra System?

<p>It limits the diffusion of signals and insulin (C)</p> Signup and view all the answers

The collaboration with Gore aims to facilitate the vascularization of the Encaptra System.

<p>True (A)</p> Signup and view all the answers

What is a critical enabling component for the Encaptra System?

<p>Semi-permeable membrane</p> Signup and view all the answers

Flashcards

Blastocysts & embryonic stem cells

Blastocysts can be used to obtain embryonic stem cells from nonviable embryos.

Removing a cell from a 2-3 day embryo

A single cell can be removed from a 2-3 day old embryo without harming the remaining cells.

Down Syndrome (chromosome 21)

A chromosomal nondisjunction event can lead to an extra chromosome 21, causing Down Syndrome.

Trisomy cause

Trisomies like Down Syndrome are due to chromosomal nondisjunction during gametogenesis (sperm or egg).

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Chromosomal Trisomy

A trisomy is when there are three copies of a chromosome instead of the normal two.

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Trisomy 13 (Patau Syndrome)

Babies with this condition rarely live into their teens, but survival is possible.

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Trisomy 18 (Edwards Syndrome)

Most babies with Edwards Syndrome die before birth or shortly after.

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Trisomy 13 & 18 frequency

Trisomy 18 occurs in 1/6000-8000 births, and Trisomy 13 in 1/8000-12000 births.

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Pregnancies to full term

30-50% of fertilized eggs are lost before implantation, 10-20% of known pregnancies end in miscarriage, most before 12 weeks; 50-70% of first-trimester miscarriages due to chromosomal abnormalities.

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Alabama Embryo Ruling

Alabama's Supreme Court ruled embryos are 'extrauterine children' in a wrongful death case related to accidental embryo destruction.

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Glucose Uptake

Glucose enters beta cells via facilitated diffusion, then phosphorylation traps it inside, preventing it from leaving.

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GLUT2

A glucose transporter protein in beta cells; it's active, but only transports glucose effectively when blood glucose levels are high.

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Potassium Ion Pumping

Increased blood glucose triggers the pumping of positively charged potassium ions out of the beta cell, changing the voltage difference across the cell membrane.

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Calcium Ion Channels

Voltage-gated channels that open when the cell's voltage changes, allowing calcium ions to flow into the beta cell.

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Insulin Release

Calcium ions in the cell cause insulin-containing granules to fuse with the cell membrane and release insulin into the bloodstream.

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Insulin Granules

Pre-loaded vesicles containing insulin. Already in beta cells and waiting to release insulin.

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Stem Cell-Derived Beta Cells

Cells created from stem cells that can produce insulin in response to high blood sugar and function like mature beta cells.

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Pluripotent Stem Cells

A type of stem cell with the ability to differentiate into a wide variety of specialized cell types, including the beta cells in the pancreas.

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Blood Glucose Levels

The concentration of glucose in the blood. Impacts insulin release.

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Threshold triggered

Increased glucose levels in blood triggers a specific mechanism for insulin release, when the level goes over 5.5 nmol/l.

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Encaptra System

A system designed to protect PEC-01 cells from the recipient's immune system, eliminating the need for immune-suppressing drugs in transplants.

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Semi-permeable membrane

A membrane that allows specific substances (like oxygen and nutrients) to pass through while blocking others.

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Vascular network formation

The creation of blood vessels on a surface, crucial for the delivery of nutrients to cells.

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Expanded PTFE composite

A material used in the Encaptra System's membrane, similar to Gore's implantable medical products.

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Fibrosis

Formation of excessive fibrous tissue (scar tissue) in an organ or tissue.

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Foreign body response

The body's immune reaction to an implanted medical device, often leading to scar tissue formation.

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PEC-01 cells

Cells protected by the Encaptra System; likely important for some function.

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Clinical trial problems

Scar tissue formation around the Encaptra implant, limiting diffusion of signals and hormones.

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Stem cell transformation to nerve cells

Converting stem cells into functional nerve cells.

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BMSC Transplantation Conditions

Conditions treatable with Bone Marrow Stem Cell (BMSC) transplantation include acute and chronic leukemias, sickle cell disease, aplastic anemia, congenital immunodeficiency diseases, lymphomas, metabolic diseases of childhood, myelodysplasia, and thalassemia.

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Leukemia & BMT

Patients with acute myeloid or lymphoblastic leukemia may benefit from bone marrow transplantation (BMT).

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First Remission BMT Success

Patients with acute myeloid leukemia in first remission after BMT have a 50-60% chance of long-term survival without disease.

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Remission Status & Relapse

The chance of relapse after BMT correlates with the patient's remission status, ranging from 20% in first remission to 60% with more advanced disease.

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Chronic Myelocytic Leukemia & BMT

Patients with chronic myelocytic leukemia in remission who receive BMT have a 60-70% chance of long-term survival.

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Pediatric BMT Expansion

Pediatric Bone Marrow Transplantation (BMT) is becoming more common due to potential cures for genetic diseases in children.

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HLA Matching

To minimize rejection, the donor and recipient's Human Leukocyte Antigen (HLA) complex should have nearly identical gene variants.

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HLA Allele Complexity

There are many known alleles for HLA-A, HLA-C, and HLA-D. However, these genes are often inherited as a unit on the same chromosome.

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Donor Scarcity

The limited availability of donors is a key challenge limiting the use of BMT.

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Alpha Cell Glucose Sensor

The alpha cell's glucose sensor, composed of GLUT1 and glucokinase, detects lower blood sugar levels than the beta cell's sensor. It triggers a response when glucose levels drop.

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Beta Cell Glucose Sensor

The beta cell's glucose sensor reacts to rising blood glucose levels, initiating insulin secretion.

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GLUT1

A glucose transporter protein in the alpha cell that transports glucose into the cell at lower blood glucose levels.

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GLUT1 Deficiency Syndrome

A genetic disorder affecting the nervous system, characterized by reduced glucose transport, especially to the brain, often resulting in seizures, developmental delays, and microcephaly.

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Glucokinase

A key enzyme in glucose metabolism that facilitates glucose entry and glycolysis in alpha cells.

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Glucose (role in brain)

Glucose is the primary fuel source and energy supply for the brain.

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Blood Glucose Levels

The concentration of glucose in the bloodstream. Crucial for energy throughout the body, including the brain.

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Insulin Secretion

Release of insulin to regulate blood sugar levels.

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Glucagon Secretion

Release of glucagon to regulate blood sugar levels, often in response to low blood sugar levels.

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Type 1 Diabetes

A chronic condition where the pancreas produces little or no insulin.

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Type 2 Diabetes

A condition where the body becomes resistant to insulin or doesn't make enough.

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Diabetes Mellitus

A group of metabolic diseases with high blood glucose.

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High Blood Glucose

Elevated blood sugar levels, a key characteristic of diabetes.

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Polyuria

Frequent urination, a symptom of high blood sugar.

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Polydipsia

Increased thirst, a symptom of high blood sugar.

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Polyphagia

Increased hunger, a symptom of high blood sugar.

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Insulin

A hormone that helps cells absorb glucose for energy.

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Spasticity

Stiffness caused by abnormal muscle tension.

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Ataxia

Difficulties coordinating movements.

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Dysarthria

Difficulties with speech.

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Myoclonus

Muscle twitches.

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Lethargy

Lack of energy.

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Fasting

Time without food or drink.

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ESC

Embryonic Stem Cells, harvested from the inner cell mass of a blastocyst.

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ASC

Adult Stem Cells, found in various adult tissues.

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iPSC

Induced Pluripotent Stem Cells, reprogrammed adult cells to become pluripotent.

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Stem Cell Self-Renewal

Stem cells' ability to divide and make identical copies of themselves.

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Stem Cell Differentiation

The process by which stem cells become specialized cells.

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Pluripotent Stem Cells

Stem cells able to differentiate into any cell type in the body.

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Totipotent Stem Cells

Early embryonic cells with the potential to form a complete organism.

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Multipotent Stem Cells

Stem cells differentiated, but able to form several cell types within a tissue lineage or organ type.

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Embryonic Stem Cell Source

Harvested from the inner cell mass of a blastocyst.

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Fetal Stem Cell Source

Derived from gonadal tissues of aborted fetuses.

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Umbilical Cord Stem Cell Source

Stem cells from umbilical cord blood.

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Placenta Derived Stem Cell Source

Stem cells from the placenta.

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Embryonic stem cells

Cells derived from an early-stage embryo (blastocyst) that can differentiate into various specialized cell types.

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Non-viable embryos

Embryos that have not developed normally and are not expected to develop into a healthy fetus.

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Embryonic cell lines

Cultured cells derived from the inner cell mass of a disrupted embryo, able to differentiate.

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In vitro fertilization (IVF)

A process where eggs are fertilized by sperm outside the body, often resulting in multiple embryos.

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Morphological criteria

Classification of embryos based on their physical characteristics under a microscope.

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Blastocyst

A stage of early embryonic development, characterized by a hollow ball of cells with an inner cell mass.

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Inner cell mass

The inner portion of a blastocyst, which will eventually develop into the fetus.

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Chromosomal trisomy

A genetic condition where a chromosome has three copies instead of the usual two.

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Genetic defect

A fault or abnormality in a gene or chromosome that can lead to a health problem.

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Encaptra System

A system protecting PEC-01 cells from the recipient's immune system, eliminating the need for immunosuppressants in transplants.

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Semi-permeable membrane

A membrane allowing some substances to pass through while blocking others.

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Vascular network formation

The growth of blood vessels on a surface, needed to deliver nutrients to cells.

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Expanded PTFE composite

A material used in the Encaptra System's membrane, similar to Gore's products.

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Fibrosis

The formation of excess fibrous tissue (scar tissue) in an organ or tissue.

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Foreign body response

The immune system's reaction to an implanted device, often causing scar tissue formation.

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PEC-01 cells

Cells protected by the Encaptra System, likely important for function.

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Clinical trial problems

Scar tissue formation around the implant, hindering signal and hormone diffusion.

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Stem cell transformation

Converting stem cells into functional nerve cells.

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Blastocysts and embryonic stem cells

Blastocysts, produced from embryos, provide a route to embryonic stem cells, especially from nonviable embryos.

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Removing a cell from an embryo

A single cell can be removed from a 2-3 day-old embryo without negatively impacting the remaining cells' viability.

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Down Syndrome (chromosome 21)

A chromosomal nondisjunction event (in gamete formation) resulting in an extra copy of chromosome 21, causing Down Syndrome.

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Trisomy cause

Trisomies result from chromosomal nondisjunction during gametogenesis (sperm or egg production).

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Chromosomal Trisomy

A chromosomal trisomy involves the presence of three copies of a specific chromosome instead of the usual two.

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Trisomy 13 (Patau Syndrome)

Babies with Trisomy 13 (Patau Syndrome) rarely live into their teens, though some can survive for a time.

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Trisomy 18 (Edwards Syndrome)

Most babies with Trisomy 18 (Edwards Syndrome) die before birth or soon after birth due to severe heart/lung defects.

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Trisomy 13 & 18 frequency

Trisomy 18 occurs in approximately 1 in 6,000-8,000 live births, and Trisomy 13 in 1 in 8,000-12,000 live births.

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Pregnancies completed

A significant percentage of fertilized eggs are lost before/during implantation, and 10-20% of known pregnancies end in miscarriage, often before 12 weeks; chromosomal abnormalities frequently observed.

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Alabama Embryo Ruling

Alabama's Supreme Court ruled that embryos are considered 'extrauterine children' in a wrongful death lawsuit involving a fertility clinic and hospital.

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Acute Myelogenous Leukemia

A type of cancer affecting blood-forming cells.

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Bone Marrow Donor

A person donating blood-forming stem cells to potentially cure a patient with blood cancer.

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HLA complex

A group of genes on chromosomes that determine proteins involved in immune function.

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Hematopoietic stem cells (HSCs)

Cells capable of developing into various types of blood cells.

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Insulin release

The process of releasing insulin into the bloodstream to regulate blood sugar.

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Voltage-gated ion channels

Channels in cell membranes that open and close in response to voltage changes.

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Calcium ion influx

The movement of calcium ions into a cell.

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Pancreatic beta cells

Cells in the pancreas responsible for insulin secretion.

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Mechanosensitive ion channels

Channels that open in response to mechanical stimuli.

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BMSC Transplantation Conditions

Conditions treatable with Bone Marrow Stem Cell (BMSC) transplantation include acute and chronic leukemias, sickle cell disease, aplastic anemia, congenital immunodeficiency diseases, lymphomas, metabolic diseases of childhood, myelodysplasia, and thalassemia.

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Leukemia & BMT

Patients with acute myeloid or lymphoblastic leukemia may benefit from bone marrow transplantation (BMT).

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First Remission BMT Success

Patients with acute myeloid leukemia in first remission after BMT have a 50-60% chance of long-term survival without disease.

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Remission Status & Relapse

The chance of relapse after BMT correlates with the patient's remission status, ranging from 20% in first remission to 60% with more advanced disease.

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Chronic Myelocytic Leukemia & BMT

Patients with chronic myelocytic leukemia in remission who receive BMT have a 60-70% chance of long-term survival.

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Pediatric BMT Expansion

Pediatric Bone Marrow Transplantation (BMT) is becoming more common due to potential cures for genetic diseases in children.

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HLA Matching

To minimize rejection, the donor and recipient's Human Leukocyte Antigen (HLA) complex should have nearly identical gene variants.

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HLA Allele Complexity

There are many known alleles for HLA-A, HLA-C, and HLA-D. However, these genes are often inherited as a unit on the same chromosome.

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Donor Scarcity

The limited availability of donors is a key challenge limiting the use of BMT.

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Study Notes

Embryonic Stem Cells

  • Embryonic stem cells (ESCs) are harvested from the inner cell mass of the blastocyst, a stage of early embryo development.
  • ESCs are pluripotent, meaning they can develop into any cell type in the body.
  • ESCs are harvested from embryos created for in vitro fertilization (IVF).

Adult Stem Cells

  • Adult stem cells are found in various adult tissues.
  • Adult stem cells are multipotent, meaning they can differentiate into a limited range of cell types.
  • Sources of adult stem cells include bone marrow, umbilical cord blood, and placenta.

Induced Pluripotent Stem Cells (iPSCs)

  • iPSCs are derived from adult cells, usually skin cells.
  • Scientists reprogram adult cells to mimic the properties of embryonic stem cells.
  • Reprogramming typically uses a combination of transcription factors.

Stem Cell Types

  • Totipotent: Each cell can develop into a new individual. Example: cells from early embryos (1-3 days).
  • Pluripotent: Capable of differentiating into any cell type (over 200 cell types). Example: Some cells of blastocyst (5 to 14 days).
  • Multipotent: Capable of differentiating into a limited number of cell types. Example: Fetal tissue, cord blood, and adult stem cells

Stem Cell Applications

  • Stem cells can be used to study development and disease.
  • Stem cells can potentially replace damaged or diseased cells in the body.
  • Stem cells may be used in regenerative medicine.

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