Gene therapy

Gene therapy

Gene therapy is the therapeutic delivery of nucleic acid in a patient’s cells as a drug to treat disease. [1] The first attempt at human-modifying DNA Was Performed in 1980 by Martin Cline , drank the first successful nuclear gene transfer in humans, approved by the National Institutes of Health , Was Performed in May 1989. [2] The first therapeutic use of gene transfer as well as the first live …

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Voretigene neparvovec

Voretigene neparvovec ( Luxturna ) is a novel gene therapy for the treatment of Leber’s congenital amaurosis . It was developed by Spark Therapeutics and Children’s Hospital of Philadelphia . [1] [2] It is the first in vivo gene therapy approved by the FDA. [3]

Synthetic rescue

Synthetic rescue (or synthetic recovery or synthetic viability when a lethal phenotype is rescued [1] ) refers to a genetic interaction in which a nonviable or sensitive to a specific drug due to the presence of a genetic mutation becomes viable when the original mutation is combined with a second mutation in a different gene. [2] The second mutation can be a loss-of-function mutation (equivalent to …

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Strimvelis is the first ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID (Severe Combined Immunodeficiency due to Adenosine Deaminase Deficiency), a rare disorder caused by the absence of an essential protein called adenosine deaminase (ADA ), which is required for the production of lymphocytes . Children born with ADA-SCID do not develop a healthy immune system so we can not fight off …

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Photochemical internalization

Photochemical internalization (PCI) is a drug and gene therapy delivery method that is designed to improve the release of macromolecules and hydrophilic chemotherapeutic agents from endosomes and lysosomes to the cytosol of targeted cancer cells . PCI is based on the use of endosomal and lysosomal Localizing amphiphilic photosensitizers qui, after-activation by light , Induce photochemical reactions resulting and in destruction of endocytic membrane mediated by reactive oxygen species (ROS). Teaphotochemical destabilization of the membrane of the endocytic vesicle result in an endosomal …

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Neovasculgen is a gene-therapy drug for treatment of peripheral artery disease , including critical limb ischemia ; it delivers the gene encoding for vascular endothelial growth factor(VEGF). [1] [2] Neovasculogen is a plasmid encoding the CMV promoter and the amino acid 165 form of VEGF. [3] [4] It was developed by the Human Stem Cells Institute in Russia and approved in Russia in 2011.

Mitochondrial replacement therapy

Mitochondrial replacement ( MRT , sometimes called mitochondrial donation ) is a special form of in vitro fertilization in which the future baby’s mitochondrial DNA comes from a third party. This technique is used when mothers carry genes for mitochondrial diseases . The two most common techniques in mitochondrial donation are pronuclear transfer and maternal spindle transfer .

Katherine A. High

Katherine A. High is an American doctor who is an Emeritus Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania . She is currently the President and Chief Scientific Officer of Spark Therapeutics . High earned at AB in chemistry at the Harvard College in 1972 and a MD in 1978 at the University of North Carolina School of Medicine . She did her training in hematology with Edward J. Benz, Jr. at Yale University . [1]