Gene therapy

Gene therapy

Gene Therapy uses genes to treat or prevent disease. Types of gene therapy include replacing a mutated gene with a healthy copy, inactivating or knocking out a mutated gene or introducing a new gene to fight disease.

Gene Therapy uses genes to treat or prevent disease. Types of gene therapy include replacing a mutated gene with a healthy copy, inactivating or knocking out a mutated gene or introducing a new gene to fight disease. In vivo gene therapy approaches directly introduce the gene therapy into the body. Ex vivo approaches in gene therapy first remove cells from the patient and genetic modification occurs outside the body, followed by reintroduction of cells back into the patient.

In vivo gene therapy

HITI method

A CRISPR/Cas9-based strategy, termed homology-independent targeted insertion (HITI) enables targeted gene insertion in non-dividing cells, both in vitro and in vivo. This technique was developed to overcome a barrier to gene therapy in that many technologies are not able to target non-dividing cells such as neurons.

Timeline

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November 5, 2020
BioSpace
Taysha Gene Therapies Bolsters Manufacturing Capacity Through Partnership with Catalent - read this article along with other careers information, tips and advice on BioSpace
BioSpace
May 15, 2020
BioSpace
Orchard Therapeutics, a global gene therapy leader, announced new interim data from an ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203, an ex vivo autologous hematopoietic stem cell gene therapy in development for the treatment of mucopolysaccharidosis type I at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy.
BioSpace
May 11, 2020
BioSpace
Atara Biotherapeutics, Inc., a pioneer in T-cell immunotherapy leveraging its novel allogeneic EBV T-cell platform to develop transformative therapies for patients with severe diseases including solid tumors, hematologic cancers and autoimmune diseases, announced the appointment of immunology and cell & gene therapy expert Maria Grazia Roncarolo, MD, to the Board of Directors.
Research and Markets
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www.prnewswire.com:443
DUBLIN, Feb. 18, 2019 /PRNewswire/ -- The "Gene Therapy - Technologies, Markets & Companies" report from Jain PharmaBiotech has been added to...

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