Oncology & Cancer
Consumer Health: Understanding thyroid cancer risk
January is Thyroid Awareness Month, which makes this a good time to learn about thyroid cancer risk factors.
Jan 26, 2022
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News tagged with genetic therapy
Genetics
Gene therapy for the treatment of Huntington's disease
Gene therapy targeting the messenger RNA (mRNA) of the mutated huntingtin gene (HTT) can provide long-lasting therapeutic benefit in Huntington's disease after a single administration. An adeno-associated virus (AAV) gene ...
Jan 25, 2022
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Genetics
New gene therapy approach offers a potential long-term treatment for limb-girdle muscular dystrophy 2B
Children's National Hospital experts developed a new pre-clinical gene therapy for a rare disorder, known as limb-girdle muscular dystrophy (LGMD) 2B, that addresses the primary cellular deficit associated with this disease. ...
Jan 4, 2022
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Genetics
Determining the minimally effective dose for gene therapy in hemophilia A
Researchers determined the minimally effective dose (MED) of a clinical candidate gene therapy vector for treating hemophilia A. This pharmacology study, performed in a mouse model and designed to support the initiation of ...
Dec 16, 2021
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Medications
First in-human study of golodirsen for Duchenne muscular dystrophy shows safety and biologic activity
This first-in-human study of golodirsen showed its long-term safety and biologic activity in patients with Duchenne Muscular Dystrophy (DMD). The approved exon-skipping therapy is designed to enable the production of functional ...
Nov 30, 2021
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Genetics
Gene therapy may reverse Hurler syndrome, a rare and severe illness in kids
Gene therapy might soon offer a new option for children with a rare genetic disorder that damages tissues throughout the body, researchers are reporting.
Nov 18, 2021
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Ophthalmology
The hunt for viruses to cure blindness
A novel computational platform developed by researchers from the University of Pittsburgh School of Medicine identifies top-performing viral vectors that could deliver gene therapies to the retina with maximum efficiency ...
Oct 19, 2021
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Neuroscience
Study suggests genetic disease CDKL5 deficiency disorder could be treatable after childhood
A devastating genetic disease called CDKL5 deficiency disorder (CDD), which strikes in early childhood, may be significantly treatable even in adulthood, a new study from the Perelman School of Medicine at the University ...
Oct 15, 2021
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Genetics
Study: Genetic kidney disorder reversible in preclinical models
Autosomal dominant polycystic kidney disease (ADPKD), a genetic disorder, causes fluid-filled cysts to develop on the kidneys, which can impair their function. As part of the growth of cysts, the kidneys develop inflammation ...
Oct 13, 2021
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Oncology & Cancer
Epigenetics predicts the efficacy of T-lymphocyte treatments in hematological malignancies
In the last few years, an alternative for these cases has emerged: a cell therapy known as CAR-T that collects the T-lymphocytes of these patients, modifies them through genetic engineering in the laboratory and administer ...
Oct 4, 2021
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