Precision gene therapy for liver disorders increases number of correctly repaired cells
Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...
Genetic mechanisms underlying Duchenne muscular dystrophy point to potential treatments
Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The ...
GBH11h
His genes forecast Alzheimer's. His brain had other plans.
Doug Whitney was supposed to develop Alzheimer's by 50. Now scientists are trying to understand why his brain remains healthy ...
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Revealing the Role of DNA Repair Genes in Huntington's
Huntington's disease is a neurodegenerative disorder that is usually fatal about 15 to 20 years after a patient is diagnosed. It is known ... | Genetics And Genomics ...
New study reveals how DNA repair genes play a major role in Huntington's disease
A new UCLA Health study has discovered in mouse models that genes associated with repairing mismatched DNA are critical in eliciting damages to neurons that are most vulnerable in Huntington's disease ...
Sickle Cell Disease News1d
SCD patients can access Casgevy via England’s public health system
Vertex has entered a reimbursement agreement with NHS England enabling eligible SCD patients to access Casgevy in the public ...
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Sickle Cell Anemia Testing & Screening Market to Reach USD 383 Million by 2030, Growing at a CAGR of 6.1%
Understanding Sickle Cell Anemia and the Need for Early DiagnosisSickle cell anemia is a genetic blood disorder caused by a ...
MedPage Today5d
Gene Therapy May Be a Game Changer for Transfusion-Dependent Thalassemia
The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...