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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.

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BioSpace
June 23, 2021
BioSpace
Immusoft, a cell therapy company dedicated to improving the lives of patients with rare diseases, announced today the formation of its Scientific Advisory Board (SAB) composed of world-renowned experts to provide external scientific review and high-level counsel on the Company's research and development programs.
BioSpace
May 24, 2021
BioSpace
Kiromic Biopharma, Inc. (Nasdaq: KRBP), an immuno-oncology target discovery and gene-editing company with a proprietary artificial intelligence neural network platform (Diamond AI) that is used to develop novel oncology therapeutics, today announced the submission of a novel Investigational New Drug (IND) to the U.S. Food and Drug Administration (FDA) for a Phase 1 clinical trial
Juniper Biologics
April 15, 2021
www.prnewswire.com:443
/PRNewswire/ -- Juniper Biologics, a science-led healthcare company focused on researching, developing & commercialising novel therapies, has announced the...
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.
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