Human Gene Therapy and Stem Cell Administration

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18 Questions

What is one of the radical techniques mentioned in the text for curing autoimmune diseases?

Replacing the patient's immune system completely

How many people have received the radical immune system replacement treatment according to the text?

1,500

What was the outcome for about one third of the participants who received the immune system replacement treatment?

Continuous remission without needing further treatments

Why do doctors use intense chemotherapy and radiation before bone marrow transplants in immune system replacement treatment?

To destroy the existing immune system

Which autoimmune diseases were specifically mentioned in the text as examples of those that can have tragic effects?

Multiple sclerosis and scleroderma

What is a common effect of autoimmune diseases on patients?

Relatively normal life expectancy

What is the main reason why each B cell only produces one kind of antibody?

Antibody genes are rearranged randomly in each B cell

What was the problem Georges Köhler and César Milstein's technique aimed to solve?

Inability to clone lymphocytes continuously in culture

What is the key factor that allows hybridoma cells to produce homogeneous monoclonal antibodies?

Cloning of the hybridoma cells in culture

Why is it not possible for each B cell to be a direct source of homogeneous antibodies by cloning?

Limitations in the growth of lymphocytes in culture

Which aspect enabled the production of antibodies to almost any antigen in quantity using monoclonal antibodies?

Cloning hybridoma cells for homogeneous antibody production

What distinguishes the resulting hybridoma cells from normal lymphocytes?

Capability to be cloned continuously in culture

What is the primary vector used in ex vivo gene therapy for SCID?

Maloney murine leukemia virus (MMLV)

Why are the gag, pol, and env genes removed from the MMLV used in gene therapy?

To prevent viral replication

In ex vivo gene therapy for SCID, what happens to the MMLV particles after they infect the packaging cell line?

They are collected for use in infecting target cells

What is the main purpose of using controls in ex vivo gene therapy with MMLV?

To prevent mutated viruses from escaping to other tissues

Which method of human gene therapy involves removing somatic cells from the patient for manipulation?

Ex vivo gene therapy

What is the main target of infection in ex vivo gene therapy for SCID using MMLV?

Bone marrow cells

Explore the process of human gene therapy, including the administration of bone marrow stem cells in addition to T cells. Learn about the two types of delivery methods, with a focus on ex vivo delivery used to combat Severe Combined Immunodeficiency (SCID).

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