effectively delivering gene-targeting drugs to the brain has remained an elusive goal. Against this backdrop, a research team from Tokyo University of Science (TUS), Japan, led by Professor Makiya ...

Getting therapeutic drugs past the blood-brain barrier has long been a major challenge in treating brain diseases. Now, researchers have explored how cholesterol-modified heteroduplex oligonucleotides ...

Hathway’s technology uses natural epigenetic processes to dial gene activity up or down, rather than rewriting the genetic code. The company is using this to develop therapies for rare diseases and ...

a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the ... Oct. 28, 2024 ...

today reported new data from its two preclinical programs targeting tau for the treatment of Alzheimer’s disease (AD). Data on tau silencing gene therapy VY1706 and anti-tau antibody VY7523 will ...

Single IV administration of tau silencing gene therapy VY1706 significantly reduced tau mRNA and protein levels, with broad brain distribution and liver de-targeting, in NHP study - - Preclinical ...