Advances in stem cell transplantation and gene therapy have been pioneered in vision research. An international team of researchers from Bristol, Toronto, Pittsburgh, Dallas and Montreal have identified a gene that could ...
Jun 10, 2015
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Gene therapy for Leber congenital amaurosis (LCA), an inherited disorder that causes vision loss starting in childhood, improved patients' eyesight and the sensitivity of the retina within weeks of treatment. Both of these ...
May 3, 2015
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In a small study that included seven children and teens with Wiskott-Aldrich syndrome, a rare immunodeficiency disorder, use of gene therapy resulted in clinical improvement in infectious complications, severe eczema, and ...
Apr 21, 2015
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UCLA stem cell researchers have pioneered a stem cell gene therapy cure for children born with adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID), often called "Bubble Baby" disease, a life-threatening ...
Nov 20, 2014
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A new form of gene therapy for boys with X-linked severe combined immunodeficiency syndrome (SCID-X1), a life-threatening condition also known as "bubble boy" disease, appears to be both effective and safe, according to a ...
Oct 8, 2014
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Every day, organ transplant patients around the world take a drug called rapamycin to keep their immune systems from rejecting their new kidneys and hearts. New research suggests that the same drug could help brain tumor ...
Sep 29, 2014
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For the first time in the world, a patient with a mechanical heart pump has taken part in a new gene therapy trial for heart failure at Harefield Hospital, London.
Aug 11, 2014
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Between 30 and 40 million people in Europe suffer from rare diseases—many of them children. As most of these diseases have genetic origins, gene therapy is a major hope for their future cure. Until now, however, there ...
Jul 2, 2014
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(Medical Xpress)—Researchers reported promising outcomes data for the first group of boys with X-linked severe combined immunodeficiency syndrome (SCID-X1), a fatal genetic immunodeficiency also known as "bubble boy" disease, ...
Dec 9, 2013
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Results from a clinical trial of eteplirsen, a drug designed to treat Duchenne muscular dystrophy, suggest that the therapy allows participants to walk farther than people treated with placebo and dramatically increases production ...
Aug 8, 2013
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