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  2. Gene therapy of the human retina - Wikipedia

    en.wikipedia.org/wiki/Gene_therapy_of_the_human...

    Retinal gene therapy holds a promise in treating different forms of non-inherited and inherited blindness. In 2008, three independent research groups reported that patients with the rare genetic retinal disease Leber's congenital amaurosis had been successfully treated using gene therapy with adeno-associated virus (AAV).

  3. California Institute for Regenerative Medicine - Wikipedia

    en.wikipedia.org/wiki/California_Institute_for...

    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.

  4. Gene therapy for color blindness - Wikipedia

    en.wikipedia.org/wiki/Gene_therapy_for_color...

    The first retinal gene therapy to be approved by the FDA was Voretigene neparvovec in 2017, which treats Leber's congenital amaurosis, a genetic disorder that can lead to blindness. These treatments also use subretinal injections of AAV vector and are therefore foundational to research in gene therapy for color blindness. [2] [3]

  5. Center for Cell and Gene Therapy - Wikipedia

    en.wikipedia.org/wiki/Center_for_Cell_and_Gene...

    The Center for Cell and Gene Therapy conducts research into numerous diseases, including but not limited to pediatric cancers, [5] [6] diabetes, [7] HIV, glioma [8] and cardiovascular disease. The center has laboratory space in both Baylor College of Medicine and Texas Children's Hospital, and clinical units in Texas Children's and Methodist ...

  6. City of Hope National Medical Center - Wikipedia

    en.wikipedia.org/wiki/City_of_Hope_National...

    City of Hope is a private, non-profit clinical research center, hospital and graduate school located in Duarte, California, United States.The center's main campus resides on 110 acres (45 ha) of land adjacent to the boundaries of Duarte and Irwindale, with a network of clinical practice locations throughout Southern California, satellite offices in Monrovia and Irwindale, and regional ...

  7. List of gene therapies - Wikipedia

    en.wikipedia.org/wiki/List_of_gene_therapies

    Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase deficiency (no longer commercially available); Axicabtagene ciloleucel (Yescarta): treatment for large B-cell lymphoma [1]

  8. Current Gene Therapy - Wikipedia

    en.wikipedia.org/wiki/Current_Gene_Therapy

    Current Gene Therapy is a peer-reviewed medical journal published by Bentham Science Publishers.The editor-in-chief is Liang Cheng (Harbin Medical University Harbin, China). ). The focus of this journal is pre-clinical or clinical research on gene the

  9. Leber's hereditary optic neuropathy - Wikipedia

    en.wikipedia.org/wiki/Leber's_hereditary_optic...

    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 ...

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