Gene Therapy

Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene in order to replace a mutated gene. Other forms involve directly correcting a mutation, or using DNA that encodes a therapeutic protein drug (rather than a natural human gene) to provide treatment. In gene therapy, DNA that encodes a therapeutic protein is packaged within a "vector", which is used to get the DNA inside cells within the body. Once inside, the DNA becomes expressed by the cell machinery, resulting in the production of therapeutic protein, which in turn treats the patient's disease.

Gene therapy was first conceptualized in 1972, with the authors urging caution before commencing gene therapy studies in humans. The first FDA-approved gene therapy experiment in the United States occurred in 1990, when Ashanti DeSilva was treated for ADA-SCID. Since then, over 1,700 clinical trials have been conducted using a number of techniques for gene therapy.

Although early clinical failures led many to dismiss gene therapy as over-hyped, clinical successes in 2006-2011 have bolstered new optimism in the promise of gene therapy. These include successful treatment of patients with the retinal disease Leber's congenital amaurosis, X-linked SCID, ADA-SCID, adrenoleukodystrophy, chronic myelogenous leukemia (CML), and Parkinson's disease. These recent clinical successes have led to a renewed interest in gene therapy, with several articles in scientific and popular publications calling for continued investment in the field.

Read more about Gene Therapy:  Approach, Vectors in Gene Therapy, Problems, Preventive Gene Therapy, In Popular Culture

Other articles related to "gene therapy, gene, genes, therapy":

Gene Therapy - In Popular Culture
... In the TV series Dark Angel gene therapy is mentioned as one of the practices performed on transgenics and their surrogate mothers at Manticore, and in the episode Prodigy, Dr ... Tanaka uses a groundbreaking new form of gene therapy to turn Jude, a premature, vegetative baby of a crack/cocaine addict, into a boy genius ... Gene therapy is a crucial plot element in the video game Metal Gear Solid, where it has been used to illegally enhance the battle capabilities of soldiers within ...
Molecular Cloning - Production of Recombinant Proteins - Gene Therapy
... Gene therapy involves supplying a functional gene to cells lacking that function, with the aim of correcting a genetic disorder or acquired disease ... Gene therapy can be broadly divided into two categories ... This “germ line gene therapy” is considered by many to be unethical in human beings ...
Dependovirus - Gene Therapy
... trigger an immune response this makes it a good virus to use as a gene therapy tool ... Gene therapy is a possible treatment for a variety of disorders and diseases that are genetic in origin ... currently being developed to transport genes into human cells ...
Wolman Disease - Prognosis
... Antibiotics for infections Steroid replacement therapy because of concerns about adrenal function Investigational Therapies Some children with Wolman ... is necessary to determine the long-term safety and effectiveness of this potential therapy for infants with Wolman disease ... Researchers are currently studying enzyme replacement therapy for LSDs such as Wolman disease ...
Small Hairpin RNA - Applications in Gene Therapy
... of shRNA to provide specific, long-lasting, gene silencing there has been great interest in using shRNA for gene therapy applications ... In particular, viral based gene therapy approaches have proved dangerous in past clinical trials ... that the patient might mount an immune response against the therapy ...

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