Gene Therapy

SUNFISH: Risdiplam Meets Primary Endpoint in SMA

Risdiplam, an oral investigational agent designed to increase and sustain SMN protein levels of patients with spinal muscular atrophy (SMA), met the primary endpoint in the pivotal part 2 of...

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Beverly Davidson, PhD, Elected to the National Academy of Medicine

Beverly L. Davidson, PhD, director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics at Children's Hospital of Philadelphia, has been elected to the National Academy of Medicine....

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Faster, cheaper, better: improved gene therapy tool production

The heart of modern therapy is increasingly personalized and targeted at specific cellular or genetic deficits. This requires gene therapy, in many cases. A new study, published on 19 September...

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New gene therapy helps stroke patients develop new neurons

Ischemic stroke is a condition in which parts of the brain lose their blood supply, causing nerve damage. Once brain tissue suffers irreparable harm, the patient will experience irreversible disability...

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Nanocapsules packed with gene-editing tools could be promising platform for many gene therapies

New tools for editing genetic code offer hope for new treatments for inherited diseases, some cancers, and even stubborn viral infections. But the typical method for delivering gene therapies to...

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Medicare to Cover Breakthrough Cancer Gene Therapy

Studies have shown that CAR-T is effective against certain types of cancers, but can cause severe side effects. Source link

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Genome editing strategy opens new perspectives in the treatment of cystic fibrosis

The approach adopted by the team of the University of Trento, led by Anna Cereseto, opens new perspectives in the treatment of cystic fibrosis, a genetic disease for which no...

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CHOP Research Team Redefines the Footprint of Viral Vector Gene Therapy

Building on a track record of developing adeno-associated viral (AAV) vectors as a groundbreaking clinical tool for gene therapy and gene editing, Children's Hospital of Philadelphia (CHOP) researchers report a...

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Novel insight into microRNA function can be crucial for the development of gene therapy

Scientists at the University of Eastern Finland and the University of Oxford have shown that small RNA molecules occurring naturally in cells, i.e. microRNAs, are also abundant in cell nuclei....

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Applying the science of measurement to biology: Why bother?

by Carmen H. Coxon, Colin Longstaff, Chris Burns Both basic and translational research are continuously evolving, but the principles that underpin research integrity remain constant. These include rational, hypothesis-driven, and...

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Three signs of real progress against sickle cell disease

A decade ago, the United Nations declared June 19 to be World Sickle Cell Awareness Day- and for good reason. Source link

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Scientists pinpoint cause of fatal respiratory disorder that turns babies' lips and skin blue

Scientists used a gene editing method called CRISPR/Cas9 to generate mice that faithfully mimic a fatal respiratory disorder in newborn infants that turns their lips and skin blue. The new...

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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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