Usher Syndrome type 1F is a rare but severe genetic disease that causes deafness, lack of balance, and progressive blindness.
Apr 26, 2023
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A new study characterizes a bioengineered adeno-associated virus (AAV)3B capsid variant that demonstrates improved transduction to human liver cells. Another advantage of the AAV3B-V04 capsid was its significantly reduced ...
Apr 25, 2023
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Premature infants, especially very low birthweight babies, are at risk for intraventricular hemorrhage. A frequent complication of these brain bleeds is hydrocephalus, an accumulation of cerebrospinal fluid (CSF) in the brain ...
Apr 24, 2023
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Scientists at Trinity College Dublin today announced a significant development toward a new therapeutic treatment of glaucoma.
Apr 19, 2023
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The therapeutic potential of microtubule-associated protein targets for cancer therapy is a largely unexplored research area due to a lack of target-specific agents. Sushmita Chatterjee and a research team in nanomedicine, ...
Effective hearing loss treatments have eluded medicine because once sensory cells in the inner ear called hair cells are damaged or destroyed, they cannot be regenerated. This loss of hair cells, which can be caused by aging, ...
Apr 18, 2023
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Usher syndrome, a rare inherited genetic disease, is a leading cause of combined deafness and blindness with type 2A (USH2A) being the most common form. USH2A, caused by mutations in the USH2A gene, can include hearing loss ...
Apr 17, 2023
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A study published in the journal Stem Cell Reports on March 23, led by Dr. Ryosuke Tsuchimochi and Professor Jun Takahashi, examined the effects of combining cell transplantation and gene therapy for axonal outgrowth in the ...
Apr 10, 2023
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Autosomal dominant polycystic kidney disease (ADPKD) is the most common potentially lethal genetic disease—about a half million people in the United States alone suffer from the condition. There is no cure, but new research ...
Apr 3, 2023
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A National Institutes of Health team has identified a compound already approved by the U.S. Food and Drug Administration that keeps light-sensitive photoreceptors alive in three models of Leber congenital amaurosis type 10 ...
Mar 29, 2023
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