The authors created a useful collection of Rep protein variants with changes that alter the amino acid sequence, but these did not lead to clear improvements in how the virus worked. Instead, their ...

Frontera Therapeutics Inc. has developed FT-017, an adenovirus-associated vector(AAV)-based gene therapy that carries a human MYBPC3 optimized codon, for the treatment of hypertrophic cardiomyopathy ...

Parkinson's disease is best known for its effects on the central nervous system. In addition, recent scientific advances ...

GenScript, a global leader in life science research tools and services support, has expanded its mRNA synthesis and ...

Simoni et al. show that liver fibrosis has a negative impact on in vivo viral vector-mediated gene transfer to hepatocytes in chemically induced and genetic mouse models varying on the type and ...

Gene regulation refers to the mechanisms that act to induce or repress the expression of a gene. These include structural and chemical changes to the genetic material, binding of proteins to ...

May 29, 2025 — Scientists have documented the way a single gene in the bacterium that causes bubonic plague, Yersinia pestis, allowed it to survive hundreds of ... New AI Tool Reveals Single ...

Roche’s gene therapy unit Spark has added to its pipeline in inherited retinal diseases (IRDs) with a $216 million alliance with Spanish biotech SpliceBio. Under the terms of the deal ...

New AI Tool Reveals Single-Cell Structure of Chromosomes -- In 3D May 28, 2025 — In a major leap forward for genetic and biomedical research, scientists have developed a powerful new artificial ...

Armed with data from a small phase 1/2 bridging study, MeiraGTx is preparing to take its gene therapy for Parkinson's disease into a pivotal trial. Shares in the Anglo-US biotech rose sharply ...