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Production and Titering of Recombinant Adeno-associated Viral Vectors
08:35

Production and Titering of Recombinant Adeno-associated Viral Vectors

Published on: November 27, 2011

Promises of co-stimulatory inhibition for durable and repeatable AAV gene therapy

Klaudia Kuranda1, David Alexandre Gross2, Giuseppe Ronzitti2

  • 1Genethon, 91000 Evry, France.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|June 22, 2026
PubMed
Abstract

No abstract available in PubMed .

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
Gene Therapy00:59

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...

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