Dec. 10, 2024 — In a single IV injection, 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 ...

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 ...

Gene expression is the generation of a functional gene product from the information encoded by a gene, through the processes of transcription and translation. Gene products are often proteins ...

The company raised $200 million after early findings hinted its therapy could be more potent than others like it, including Sarepta Therapeutics’ Elevidys.

Genetic testing plays a crucial role in identifying the risk of these disorders ... Other disorders like Biotinidase deficiency, Phenylketonuria or Galactosemia can be screened using simple ...

Feb. 19, 2025 — Researchers have found a compound in ginger, called furanodienone (FDN), that selectively binds to and regulates a nuclear receptor involved in inflammatory bowel disease (IBD ...

For many Filipinos, receiving a medical diagnosis is supposed to provide clarity. But for those with rare diseases, it often ...

Additionally, almost 15,000 newborns will undergo screening for common, treatable genetic disorders, including congenital hypothyroidism, congenital adrenal hyperplasia, galactosemia, G6PD ...

The inspection took place several months before govorestat was turned down as a treatment for the rare genetic disorder ... was met with consternation by the Galactosemia Foundation, which ...

Diabetes Test Results: Types of Tests and How to Read Them If you or someone in your family has diabetes, you might be wondering if it is genetic. There are different types of diabetes. For each type, ...