Gene therapy - Wikipedia
In medicine, gene therapy (also called human gene transfer) is the therapeutic delivery of nucleic acid into a patient's cells as a drug to treat disease. The first attempt at modifying human DNA was performed in 1980 by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989.
Gene Therapy in a Patient with Sickle Cell Disease | NEJM
Sickle cell disease results from a homozygous missense mutation in the β-globin gene that causes polymerization of hemoglobin S. Gene therapy for patients with this disorder is complicated by the complex cellular abnormalities and challenges in achieving effective, persistent inhibition of polymerization of hemoglobin S. We describe our first patient treated with lentiviral vector–mediated ...
Cell And Gene Therapy Innovation Summit 2019 - Home
The Annual Cell and Gene Therapy Innovation Summit is the only gathering committed to the development of manufacturing processes for these products.
Hematopoietic Stem-Cell Gene Therapy for Cerebral ...
BackgroundIn X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function of the ALD protein. Cerebral adrenoleukodystrophy is characterized by demyelination and neurodegeneration. Disease progression, which leads to loss of neurologic function and death, can be halted only with allogeneic hematopoietic stem-cell transplantation.
CAR-T gene therapy for leukemia clears FDA hurdle - CBS News
A panel of cancer experts voted on what could become the first gene therapy widely available in the U.S.
Cellular & Gene Therapy Guidances
This page contains a listing of cellular and gene therapy guidances.
Could gene therapy become biotech’s growth driver in 2017 ...
THANK YOU Ohad ! A WONDERFUL and INSIGHTFUL summary on GENE THERAPY and viable companies! A while back you mentioned the possibility of adding FOLD.
Biotech portfolio update – Jumping into gene therapy ...
After a long summer break it is time to review recent events and update the portfolio. As far as clinical readouts go, my portfolio had a brutal summer with one complete P3 failure from Array Biopharma (), a mixed data set from Aurinia and a win from SAGE that resulted in limited share appreciation.This was offset by strong performance from Exelixis (), my biggest holding which is up 48% ...
F.D.A. Approves First Gene-Altering Leukemia Treatment ...
A technician working with human cells belonging to cancer patients at Novartis Pharmaceuticals in Morris Plains, N.J. The Food and Drug Administration on Wednesday approved Novartis’s gene therapy for leukemia, the first-ever treatment that alters a patient’s own cells to fight cancer.