Gene Therapy

More screening and new treatments needed for sickle cell disease

The promise of new treatments for sickle cell disease, a group of inherited red blood cell disorders, has never been so great, but it will only be realized if there...

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Scientists successfully carry out gene editing in vivo

Scientists at Harvard University have successfully proven that stem cells can be edited inside the body, rather than in a petri dish. Source link

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Scientists use gold nanoparticles to edit genes tied to HIV, inherited blood disorders

Scientists at Fred Hutchinson Cancer Research Center took a step toward making gene therapy more practical by simplifying the way gene-editing instructions are delivered to cells. Source link

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Newly approved gene therapy for SMA is based on delivery vehicle discovered by Penn expert

James Wilson, MD, PhD, recalls being struck by the devastating toll of rare diseases as a young physician in the 1980s. He set out on a path to correct the...

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CU Anschutz researchers identify new immunodeficiency disease

[ad_1] A new immunodeficiency disease caused by a novel genetic mutation has been identified by researchers at the University of Colorado Anschutz Medical Campus providing unique insights into cell biology....

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Experimental Agent Shows Promise in Slowing Genetic Form of ALS

Researchers successfully target elevated levels of a harmful protein, offering hope to people with a genetic form of amyotrophic lateral sclerosis. Medscape Medical News Source link

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Getting serious about the challenge of regulating germline gene therapy

by Arthur Caplan The announcement of He Jiankui’s germline editing of human embryos has been followed by a torrent of almost universal criticism of the claim on scientific and ethical...

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New gene therapy cures babies with fatal 'Bubble Boy' disease

[ad_1] A new gene therapy created by US researchers appears to have cured eight infant boys born with a rare genetic disorder referred to as “Bubble Boy” disease. Source link...

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Gene Therapy Restores Immunity for ‘Bubble Boys’

[ad_1] A team of scientists say they have treated 10 "Bubble Boy' babies born with the disease called X-linked combined severe immunodeficiency, or SCID-X1, using a combination of gene therapy...

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Tumor biomarker EpCAM shown to be promising target for cancer immunotherapy

[ad_1] Researchers have shown that cancer immunotherapy targeting the tumor biomarker epithelial cell adhesion molecule is safe and nontoxic in mice and can significantly delay tumor formation and growth. Source...

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Unique gene therapy approach paves new way to tackle rare, inherited diseases

[ad_1] Nonsense mutations are single-letter errors in the genetic code that prematurely halt the production of critical proteins. These unfinished proteins are unable to function normally, and nonsense mutations cause...

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Gene therapy restores hearing in mice with congenital genetic deafness

[ad_1] In collaboration with the universities of Miami, Columbia and San Francisco, scientists from the Institut Pasteur, Inserm, CNRS, Collège de France, Sorbonne University and the University of Clermont Auvergne...

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Oxford Genetics signs licensing agreement with Aldevron to market lentiviral packaging plasmids

[ad_1] Oxford Genetics, a leading innovator of biopharmaceutical technologies, has signed a licensing agreement with world-class service organization, Aldevron to bring to market a standardized range of its plasmids for...

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Gene Therapy Shows Promise Against Sickle Cell

[ad_1] Despite promising results, it's unclear if the effects of treatment will last and it's likely to be at least three years before a genetic therapy for sickle cell disease...

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Scientists develop new gene therapy that prevents axon destruction in mice

[ad_1] Nerve axons serve as the wiring of the nervous system, sending electrical signals that control movement and sense of touch. Source link [ad_2]

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Novel gene therapy could dampen drug-induced side effect in Parkinson's patients

[ad_1] A Michigan State University researcher has received a $2.8 million federal grant to develop a gene therapy that could reduce and possibly eliminate a frustrating side effect of a...

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New method modifies blood stem cells to reverse genetic mutation that causes IPEX

[ad_1] UCLA researchers led by Dr. Donald Kohn have created a method for modifying blood stem cells to reverse the genetic mutation that causes a life-threatening autoimmune syndrome called IPEX....

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Children's Hospital Celebrates European Commission Approval of First-of-Its-Kind Gene Therapy for Blindness

[ad_1] Children's Hospital of Philadelphia celebrates a pivotal moment in medicine: approval by the European Commission of LUXTURNA(r) (voretigene neparvovec), the first and only gene therapy for patients with an...

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Advances in gene therapy for inherited blood disorders

[ad_1] Gene therapy holds a lot of promise in medicine. If we could safely alter our own DNA, we might eliminate diseases our ancestors passed down to us. Source link...

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Sickle Cell Gene Therapy Makes Another Advance

[ad_1] The therapy targets the genetic flaw that causes sickle cell. In a small group of patients, researchers said the therapy appears safe and effective enough to keep moving forward...

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