News-Medical.Net Gene Therapy News
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Toward a transformative therapy for sickle cell patients
Apr 9, 2021 | 17:43 pmFor the millions of people worldwide who have sickle cell disease, there are only a few treatment options, which include risky bone marrow transplants, gene therapy or other treatments that address a subset of symptoms.
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Rice bioengineer wins NIH support for a new strategy to fight cystic fibrosis
Apr 9, 2021 | 01:44 amChemical and biomolecular engineer Xue Sherry Gao of Rice University's Brown School of Engineering has won National Institutes of Health support for a new strategy to fight cystic fibrosis.
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Researchers evaluate new drug for spinal muscular atrophy in a broader spectrum of patients
Apr 6, 2021 | 20:14 pmThe rarity of spinal muscular atrophy (SMA) means that promising new treatments may be tested in only a limited spectrum of patients before approval. Investigators evaluated a newly approved drug, onasemnogene abeparvovec, in a broader spectrum of patients in order to obtain expanded data on its side effects profile.
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New approach to CAR T gene therapy yields efficient and longer-lasting response against HIV
Apr 3, 2021 | 20:20 pmA UCLA research team has shown that using a truncated form of the CD4 molecule as part of a gene therapy to combat HIV yielded superior and longer-lasting results in mouse models than previous similar therapies using the CD4 molecule.
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Fight for Sight funds research to explore gene editing as treatment for inherited retinal diseases
Apr 1, 2021 | 13:51 pmFight for Sight is funding cutting-edge research at UCL, which will explore whether gene editing could be used to repair faults in genes for some inherited retinal diseases.
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Gene therapy restores vison in dogs with severe form of Leber congenital amaurosis
Apr 1, 2021 | 01:43 amGustavo Aguirre and William Beltran, veterinary ophthalmologists and vision scientists at the University of Pennsylvania School of Veterinary Medicine, have studied a wide range of different retinal blinding disorders.
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Scientists launch clinical trial of CRISPR gene correction therapy in patients with sickle cell disease
Mar 31, 2021 | 02:09 amScientists at UC San Francisco, UC Berkeley and UCLA have received U.S. Food and Drug Administration approval to jointly launch an early phase, first-in-human clinical trial of a CRISPR gene correction therapy in patients with sickle cell disease using the patient's own blood-forming stem cells.
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First cell-based gene therapy approved by the FDA for treating multiple myeloma
Mar 29, 2021 | 06:14 amThe U.S. Food and Drug Administration approved Abecma (idecabtagene vicleucel), a cell-based gene therapy to treat adult patients with multiple myeloma who have not responded to, or whose disease has returned after, at least four prior lines (different types) of therapy.
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Mogrify wins Hewitsons Award for Innovation in Business and Price Bailey Award for Business of the Year at CambridgeshireLive Business Excellence Awards 2020
Mar 26, 2021 | 11:04 amMogrify has been awarded the Hewitsons Award for Innovation in Business and Price Bailey Award for Business of the Year.
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Novel gene therapy reprograms glioma cells into functional neurons
Mar 24, 2021 | 06:13 amGlioma is a fatal neurological disorder that has limited interventional treatment, despite extensive research over the past several decades.
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