Stem Cell Research Quiz
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Questions and Answers

What is one of the main benefits of embryonic stem cells (ES cells)?

  • They can differentiate into all three germ layers. (correct)
  • They cannot proliferate indefinitely.
  • They are obtained without ethical concerns.
  • They have no potential for cell therapy.

Embryonic stem cells can only be derived from adult tissues.

False (B)

What procedure can be used to produce ES cells from the patient's own cells?

Somatic cell nuclear transfer (SCNT)

ES cells are crucial for the in vitro study of __________ processes which cannot be assessed otherwise.

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

Match the following concepts related to ES cells with their corresponding descriptions:

<p>Pluripotent = Ability to differentiate into any cell type Ethical concerns = Issues surrounding the destruction of human blastocysts Biobanking = Creation of an ES cell bank for immune matching Immune rejection = Potential response due to unmatched HLA haplotypes</p> Signup and view all the answers

Who received the Nobel Prize in Physiology or Medicine in 2012 for discovering that mature cells can be reprogrammed to become pluripotent?

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

Induced Pluripotent Stem Cells (iPS cells) can only be generated from embryonic stem cells.

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

What significant contribution did Shinya Yamanaka make in 2006 regarding somatic cells?

<p>He demonstrated that fully differentiated somatic cells can be reprogrammed to a pluripotent stem cell state.</p> Signup and view all the answers

Somatic cell nuclear transfer (SCNT) is a technique associated with the cloning of __________.

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

Match the following researchers with their contributions to stem cell research:

<p>John Gurdon = Nobel Prize for cell reprogramming Shinya Yamanaka = Discovery of iPS cells Colman and Kind = Research on therapeutic cloning Takahashi et al. = Induction of iPS cells from fibroblasts</p> Signup and view all the answers

What is a key advantage of induced pluripotent stem cells (iPS cells) over embryonic stem cells?

<p>They are individual-specific and non-immunogenic. (C)</p> Signup and view all the answers

List two types of somatic cells that can be used to generate iPS cells.

<p>Fibroblasts and keratinocytes.</p> Signup and view all the answers

One of the benefits of iPS cells is that there are no ethical issues involved in their use.

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

Which of the following is NOT included in the iPSC quality control checklist?

<p>Gene expression profiling (C)</p> Signup and view all the answers

The stem cell-based therapy for Parkinson's Disease is called STEM-PD.

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

What primary cells were used in the pioneering procedure for Parkinson's Disease treatment?

<p>Fetal midbrain cells</p> Signup and view all the answers

One method discussed for efficiently delivering cells is ______.

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

Match the methods of delivering cells to their descriptions:

<p>Transdifferentiation = Conversion of one cell type to another without reverting to a stem cell stage Organoids = Miniaturized and simplified organs produced in vitro Bioengineering = Using engineering principles to develop biological products Stem-PD = Stem cell-based therapy for Parkinson's Disease</p> Signup and view all the answers

What is a potential advantage of using cord blood as a source of stem cells?

<p>Increased donor availability (D)</p> Signup and view all the answers

Mesenchymal stem cells (MSCs) have significant diversity in how they are described and manufactured.

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

What is one current application of mesenchymal stem cells in clinical trials?

<p>Their immune suppression properties.</p> Signup and view all the answers

Cord blood contains both mesenchymal and __________ stem cells.

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

Match the types of stem cells with their characteristics:

<p>Pluripotent stem cells = Able to differentiate into nearly any cell type Multipotent stem cells = Limited to specific lineages Anti-inflammatory properties = Used in MSCs Short-term existence = Characteristic of allogeneic MSCs</p> Signup and view all the answers

Which of the following is NOT a disadvantage of cord blood?

<p>Lower risk of GvHD (C), Reduced need for HLA matching (D)</p> Signup and view all the answers

Current MSC clinical trials do not utilize their anti-inflammatory properties.

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

What is one challenge that needs to be addressed when taking stem cell therapies to the clinic?

<p>Scarcity of stem cells.</p> Signup and view all the answers

What is a known risk associated with induced pluripotent stem cells (iPSCs)?

<p>Tumorigenicity due to incomplete silencing of oncogenic factors (D)</p> Signup and view all the answers

Multipotent stem cells are specific to specific tissues.

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

Who performed the first successful stem cell transplant?

<p>Dr. E. Donnall Thomas</p> Signup and view all the answers

IPSC-derived retinal pigment epithelium (RPE) cells required patients to wait over _____ months for surgery.

<p>10</p> Signup and view all the answers

Match the type of stem cell with its characteristic:

<p>Hematopoietic Stem Cells (HSCs) = Most widely transplanted in stem cell therapies Mesenchymal Stem Cells (MSCs) = Can differentiate into osteocytes, chondrocytes, and adipocytes Induced Pluripotent Stem Cells (iPSCs) = Risk of genomic alterations Multipotent Stem Cells = Tissue-specific</p> Signup and view all the answers

What is a major challenge of personalized medicine using iPSCs?

<p>Long wait times and high costs (A)</p> Signup and view all the answers

The Graft versus Leukemia (GvL) effect occurs in allogeneic HSC transplantation.

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

What is the significance of hematopoietic stem cells in transplantation?

<p>They replenish the recipient's hematopoietic system.</p> Signup and view all the answers

Which of the following diseases have been targeted by iPSC-derived cell therapies?

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

Pluripotent stem cells can give rise to neural stem cells.

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

How many clinical trials involving iPSC-derived cell therapy are currently registered?

<p>19</p> Signup and view all the answers

The trial plans to enroll a total of _____ patients for transplantation, starting with patients from Sweden.

<p>8</p> Signup and view all the answers

Match the following terms related to stem cells with their descriptions:

<p>iPSCs = Stem cells generated directly from adult cells ESCs = Stem cells derived from early embryos Cord blood stem cells = Stem cells derived from umbilical cord blood Neural stem cells = Stem cells that can differentiate into neural cells</p> Signup and view all the answers

ESCs and iPSCs are equivalent for transplantations as part of stem cell therapies.

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

What is a current limitation of stem cell therapies?

<p>Potential immune rejection (A)</p> Signup and view all the answers

Induced pluripotent stem cells pose no concern of immune rejection.

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

Flashcards

What are embryonic stem cells (ES cells)?

Embryonic stem cells (ES cells) are pluripotent cells that can differentiate into any cell type in the body.

What is the key characteristic of ES cells?

ES cells possess the unique ability to multiply indefinitely while preserving their pluripotent nature.

What can ES cells be used for?

ES cells can be used to create various cell types in the lab, aiding in understanding human development. Examples include neurons, blood cells, heart cells, and liver cells.

What is a major ethical concern regarding ES cells?

One hurdle is the ethical concern surrounding the use of human blastocysts, which are destroyed to obtain ES cells.

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What is a biological challenge with transplanting ES cells?

Another challenge is immune rejection, as ES cells from a donor may differ from the recipient's immune system.

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What is Somatic Cell Nuclear Transfer (SCNT)?

The process of transferring the nucleus of a somatic cell into an enucleated egg cell to create a genetically identical copy.

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Why is Dolly the sheep significant?

Dolly the sheep was the first mammal cloned from an adult cell using SCNT, demonstrating the potential for creating genetically identical copies.

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What is therapeutic cloning?

Therapeutic cloning aims to create embryonic stem cells that are genetically identical to a patient, potentially for treating diseases or injuries.

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What are induced pluripotent stem cells (iPS cells)?

Induced pluripotent stem cells (iPS cells) are created by reprogramming adult somatic cells into a pluripotent state, similar to embryonic stem cells.

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Who discovered iPS cells and when?

Shinya Yamanaka's lab in 2006 showed that fully differentiated somatic cells can be reprogrammed into a pluripotent state using specific factors.

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What are the advantages of iPS cells over ES cells?

iPS cells have the potential to overcome ethical concerns and limitations associated with embryonic stem cells, making them a promising avenue for regenerative medicine.

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What are some potential applications of iPS cells?

iPS cells can be used to create personalized cell therapies, potentially treating conditions like Parkinson's disease and diabetes.

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What is the future of iPS cell research?

iPS cell research holds immense potential for advancing our understanding of human development, disease mechanisms, and developing personalized therapies.

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What is STR analysis for iPSCs?

Short Tandem Repeat (STR) analysis is a technique used to identify and analyze variations in the number of repeated DNA sequences. It's used to check the genetic stability and integrity of iPSCs, ensuring they have the correct genetic makeup and haven't undergone any harmful changes.

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Why is karyotyping important for iPSCs?

Karyotyping is a process where chromosomes are visualized and analyzed, identifying any abnormalities or mutations. In iPSC quality control, it ensures the iPSCs have the normal number of chromosomes (46) and no structural changes affecting their potential.

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Why is viral testing necessary for iPSCs?

Viral testing is essential to ensure iPSCs are free from any contaminating viruses that could compromise their safety and efficacy. These tests help guarantee the iPSCs are safe for use.

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What are the challenges in cell delivery for iPSCs?

The transplantation of cells, such as those derived from iPSCs, requires careful consideration of how these cells will be delivered to the target area. This includes addressing technical aspects such as the correct route and ensuring efficient cell survival and engraftment.

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How does transdifferentiation circumvent the use of iPSCs?

Transdifferentiation is a process where one cell type directly transforms into another cell type without going through an intermediate stem cell stage. This approach allows for the direct conversion of existing cells into the desired ones, potentially eliminating the need for iPSCs in some cases.

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What are induced pluripotent stem cells (iPSCs)?

Induced pluripotent stem cells (iPSCs) are a type of stem cells generated from adult cells by reprogramming them to an embryonic-like state. This process involves introducing specific genes that can re-activate the pluripotency factors, allowing these cells to potentially differentiate into any cell type in the body.

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What are mesenchymal stem cells (MSCs)?

Mesenchymal stem cells (MSCs) are multipotent stem cells that have the ability to differentiate into a variety of cell types, including bone, cartilage, and fat.

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What are the key properties of MSCs?

MSCs have anti-inflammatory properties and can influence tissue repair. This makes them promising candidates for treating various diseases and injuries.

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What is a limitation of iPSCs related to differentiation?

One of the limitations of iPSCs is the potential for incomplete differentiation. This means that not all iPSCs may fully revert to an embryonic-like state and may retain characteristics of their original adult cell type, making them unsuitable for certain applications.

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What is a limitation of iPSCs related to genomic and epigenetic alterations?

Another concern with iPSCs is the potential for genomic and epigenetic alterations. These alterations may arise during the reprogramming process and can affect the cells' normal development and function. This can potentially lead to undesirable outcomes.

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What is a key limitation of allogeneic MSCs?

One challenge in using allogeneic MSCs (from a different donor) is their short lifespan in the body. The transplanted cells might not survive long enough to be effective.

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What are hematopoietic stem cells (HSCs)?

Hematopoietic stem cells (HSCs) are multipotent stem cells found in the bone marrow that can differentiate into various blood cell types.

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How are MSCs currently being used in clinical trials?

Current MSC clinical trials often utilize their immune-suppressive properties. This means they can help suppress the immune response to prevent rejection of transplanted cells.

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What is a major application of HSCs?

HSCs are a crucial component of many stem cell therapies, as they can replenish the recipient's hematopoietic system by providing healthy blood cells.

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What is a potential source of stem cells?

Cord blood is a rich source of stem cells, including both mesenchymal and hematopoietic stem cells. It offers several advantages over other sources.

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What are the advantages of using cord blood as a source of stem cells?

Cord blood has several advantages, including readily available donors, non-invasive collection, lower risk of graft-versus-host disease (GvHD), and less stringent HLA matching requirements.

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Which type of stem cells are most widely transplanted?

HSCs are most commonly transplanted in stem cell therapies, making them the most widely used type of stem cell in clinical applications.

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What is a major challenge of HSC transplantation?

One of the challenges of using HSCs for transplants is graft-versus-host disease (GVHD). This occurs when donor immune cells attack the recipient's healthy tissues, leading to complications.

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What are the disadvantages of using cord blood as a source of stem cells?

Cord blood also has some disadvantages, such as limited numbers of hematopoietic stem cells (HSCs), which are responsible for blood cell production.

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What are mesenchymal stem cells (MSCs)?

Mesenchymal stem cells (MSCs) are multipotent cells found in various tissues that can differentiate into bone, cartilage, and fat cells.

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How can we overcome the scarcity of hematopoietic stem cells (HSCs)?

Expanding hematopoietic stem cells ex vivo (outside the body) can overcome the scarcity of these cells. This involves culturing and multiplying the cells in a controlled environment to increase their numbers.

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What are stem cells?

Stem cells are special cells that can divide and renew themselves for long periods, and can also differentiate into specialized cell types.

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What are the classifications of stem cells?

Stem cells are classified based on their potential to differentiate into other cell types. Totipotent stem cells can become any cell type, including the placenta. Pluripotent stem cells can become any cell type in the body. Multipotent stem cells can become a limited range of cell types within a specific lineage. Unipotent stem cells can only differentiate into one cell type.

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How can stem cells be used therapeutically?

Stem cell therapies aim to use the regenerative potential of stem cells to repair damaged tissues or organs. This can involve transplanting stem cells directly or using them to create specific cell types for transplantation, treating various diseases.

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What are the limitations of stem cell therapies?

Current limitations for stem cell therapies include ethical concerns surrounding the source of stem cells (embryonic or otherwise), difficulties in controlling stem cell differentiation, risks of tumor formation, and immune rejection after transplantation.

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Pluripotent stem cells can give rise to neural stem cells.

True. Pluripotent stem cells, like embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs), can differentiate into neural stem cells. These cells can then develop into various neural cells in the brain.

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ESCs and iPSCs are equivalent for transplantations.

False. Although both ESCs and iPSCs are pluripotent, they have different origins and ethical implications. ESCs come from embryos, while iPSCs are reprogrammed from adult cells.

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Umbilical cord blood derived stem cells can be used directly for clinical applications.

True. Umbilical cord blood contains hematopoietic stem cells, which can differentiate into various blood cells and are used in clinical applications for treating blood disorders and cancers.

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Induced pluripotent stem cells pose no concern of immune rejection.

False. Although iPSCs are derived from an individual's own cells, they can still elicit an immune response, requiring immune suppression therapies.

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Study Notes

Learning Outcomes

  • Define stem cells
  • Classify different stem cell types
  • Describe therapeutic use of stem cells
  • Discuss current limitations of stem cell therapies

Lecture Overview: Part 1

  • What are stem cells?
  • Stem cell classification
  • Pluripotent stem cells (ES cells and iPS cells)
  • Multipotent stem cells

Lecture Overview: Part 2

  • Translation to the clinic: several considerations
  • Current applications of Stem Cell Therapies

Stem Cell Therapy: Regenerative Medicine

  • Why? Organ shortage, donor incompatibility
  • How? Repair tissues/organs, replacing cells
  • Which sources? Isolated or induced stem cells
  • Data: 103,223 people on the US national transplant waiting list (representative data)

What are Stem Cells?

  • Stem cells have two defining qualities:
    • Self-renewal
    • Differentiation into specialized mature cells
    • A diagram showing the differentiation of hematopoietic stem cells (HSCs) is included

What is the Origin of Stem Cells?

  • Stem cells develop from a fertilized egg
  • Stages shown: fertilized oocyte, morula, blastula, gastrula, embryo
  • Different types of stem cells are shown: totipotent, pluripotent, multipotent

Stem Cell Classification

  • Totipotent: Can give rise to an entire organism (e.g., zygote)
  • Pluripotent: Can give rise to all three germ layers (mesoderm, endoderm, ectoderm)
  • Multipotent: Can give rise to a limited number of cell types

Embryonic Stem Cells (ES Cells)

  • Murine ES cell lines established in 1981
  • Human ES cell lines derived in 1998
  • Can be isolated from excess IVF eggs

ES Cells and Their Potential

  • Can proliferate indefinitely, maintaining pluripotency
  • Give rise to all three germ layers: neurons, blood cells, cardiomyocytes, liver cells
  • Crucial for studying developmental processes
  • Useful for modeling diseases (e.g., genetic diseases, etc.)
  • Have significant potential for cell therapy

Limitations of ES Cells

  • Ethical concerns: human blastocysts are sacrificed
  • Allogeneic transplantation: immune rejection due to HLA haplotype mismatch
  • Steps to circumvent immune rejection:
    • Biobanking HLA-typed cell lines
    • Somatic cell nuclear transfer (SCNT)
  • Figures and data related to these considerations are presented

Somatic Cell Nuclear Transfer (SCNT)

  • A technique to produce ES cells from a patient's own cells
  • Diagrams are used to explain the process
  • The Nobel Prize in Physiology or Medicine 2012 was awarded for this discovery

Therapeutic Cloning

  • Using SCNT to produce ES cells for a patient's own use
  • Diagram illustrating the process

Induced Pluripotent Stem Cells (iPS Cells)

  • Fully differentiated somatic cells reprogrammed into pluripotent stem cells (2006)
  • Reprogramming involves introducing specific genes
  • Significant advantage because no embryos/ethical issues
  • iPS cells possess many advantages similar to ES cells
  • iPSC can be made easily, and maintained in storage

iPSCs and their Potential

  • Overcome ethical concerns of ES cells
  • Viable alternative for cell therapies
  • Versatile for various somatic cell types (blood, keratinocytes, fibroblasts)

Limitations of iPSCs

  • Risk of incomplete differentiation
  • Genomic/epigenetic alterations in iPSC-derived cells
  • Tumorigenicity due to incomplete silencing of oncogenic factors (e.g., Myc, Klf4)
  • Personalized medicine may not be realistic
  • Financial and time limitations can be significant

Multipotent Stem Cells

  • More tissue-specific
  • Harder to access and isolate
  • Locations of adult stem cells shown in diagram

Hematopoietic Stem Cells (HSCs)

  • Most widely transplanted stem cells in therapies
  • HSCs reside in the bone marrow and replenish the hematopoietic system
  • History of HSC transplantation: post-WWII, first transplant in 1956
  • Considerations: GvHD, conditioning regimens, sources (BM, MPB, UCB)
  • Allogeneic vs. autologous transplantation
  • Indications for HSC transplantation: illustrated with a table categorizing malignancies and non-malignant disorders

Mesenchymal Stem Cells (MSCs)

  • MSCs can differentiate in vitro into osteocytes, chondrocytes, and adipocytes
  • Markers for MSCs
  • Wide use in clinical trials (types and regions illustrated in diagram)
  • Anti-inflammatory properties, tissue repair
  • Short-term existence: possible allogeneic safety
  • Immune suppression properties

How to Overcome the Scarcity of Stem Cells

  • Extracting from alternate sources (e.g., umbilical cord blood)
  • Expanding hematopoietic stem cells in vitro for increased yield
  • Different methods for expanding cells shown in diagram

How to Design Protocols to Obtain Cells of Interest from Stem Cells?

  • cGMP-compliant protocol
  • Complete differentiation of all pluripotent stem cells
  • Ensuring the specific cells of interest are obtained and tested for functionality

How to Ensure the Safety of Material to be Transplanted?

  • Risks: Teratomas, insertional mutagenesis
  • Important quality control checklist considerations
  • Genomic integrity testing

How to Efficiently Deliver the Cells?

  • Bioengineering considerations (biomaterials, injectable hydrogels, cell sheets)
  • Other delivery methods discussed (organoid development, specific example of Parkinson's Disease therapy)

Blood Formation During Development

  • Stages and locations of blood cell development
  • Diagram shows the waves and locations of hematopoietic development in an embryo

Translation to the Clinic

  • Overcoming stem cell scarcity
  • Safe and efficient protocols for obtaining specific cells
  • How stem-cells are safely delivered for transplantation and integration into patients

Parkinson's Disease

  • Use of stem cells in the clinic for Parkinson's disease
  • Clinical trial for Parkinsons disease
  • Pioneering work of A. Björklund & O. Lindvall

iPSC-Derived Cell Therapy Trials

  • Recent clinical trials using iPSC-derived cell therapy
  • Diseases Targeted by iPSC therapies

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Test your knowledge on embryonic stem cells and induced pluripotent stem cells in this quiz. Discover key concepts, notable researchers, and significant breakthroughs in stem cell research, including the contributions of Nobel laureates. Challenge yourself to match terms and describe processes in this fascinating field of study.

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