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The California Institute for Regenerative Medicine (CIRM) is a state agency that supports research and education in the fields of stem cell and gene therapiesIt was created in 2004 after 59% of California voters approved California Proposition 71: the Research and Cures Initiative, [1] which allocated $3 billion to fund stem cell research in California.
A number of recent clinical trials have suggested that gene therapy could help treat diseases of the eye that cause vision loss. This Special Feature explores the current evidence and outstanding ...
Primary congenital glaucoma is classified into three subtypes: [4] true congenital glaucoma, which causes signs of increased intraocular pressure within the first month of life, infantile glaucoma, which presents between one month and three years, and; juvenile glaucoma, which becomes clinically apparent after three years of age and before age 40.
Gene therapy is a medical procedure that involves inserting genetic material into a patient's cells to repair or fix a malfunctioning gene in order to treat hereditary illnesses. Between 1989 and December 2018, over 2,900 clinical trials of gene therapies were conducted, with more than half of them in phase I. [51]
Human clinical trials are underway at GenSight Biologics (ClinicalTrials.gov # NCT02064569) and the University of Miami (ClinicalTrials.gov # NCT02161380) to examine the safety and efficacy of mitochondrial gene therapy in LHON. In these trials, participants affected by LHON with the G11778A mutation will have a virus expressing the functional ...
In December 2013 the Doheny Eye Institute entered into an exclusive, long-term affiliation agreement with the University of California Los Angeles, [17] forming the "Doheny Eye Center UCLA". Ronald E. Smith had served as Chairman of Ophthalmology at USC from 1995 to 2013, [ 18 ] and left USC to follow Doheny, and became vice chair of the ...
Clinical trials of gene therapy for sickle cell disease were started in 2014. [227] [228] In February LentiGlobin BB305, a gene therapy treatment undergoing clinical trials for treatment of beta thalassemia gained FDA "breakthrough" status after several patients were able to forgo the frequent blood transfusions usually required to treat the ...
The original issue focused on viral vectors for gene delivery vectors and use of gene therapy treatments for oncology. Current Gene Therapy was among the first gene therapy focused journals to publish on the use of Alphaviruses for gene therapy, [2] oncolytic virotherapy using adenoviral-mediated gene transfer, [3] and the use of Fas Ligand ...
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