Megan L Noonan will present her research on the role of alternative splicing in kidney disease progression, discussing how she used nanopore RNA sequencing to accurately detect and quantify ...

Currently, there are no effective treatment methods that ... already in its pipeline for select diseases amenable to splicing modulation or modulation of gene expression, Panorama is the partner ...

The scientific advancements of the past 30 years have transformed our understanding of the potential of gene ... with rare diseases still do not have access to the right care or treatment.

This has driven Rivella, Thompson, and their colleagues at CHOP to remain focused on increasing treatment choices for sickle-cell disease. As well as developing in vivo gene editing, they are ...

with some researchers and bioethicists calling for a ban on attempts to treat even imminently lethal diseases with gene-editing techniques that would modify germ cells. The move toward prohibition ...

approved in Europe to treat the rare disease adenosine deaminase deficiency, or "bubble boy syndrome." Claire Booth, MBBS, PhD, professor of gene therapy and pediatric immunology at University ...

DENVER — A drug found in almost every medicine cabinet may be a treatment for a rare genetic disease. Ibuprofen may help children with mutations in a gene called MAN1B1, a study in fruit flies ...

A double breakthrough in the war against malaria could lead to a cure for the disease which kills a child every 20 seconds. Scientists say they have cracked the genetic code of both the mosquito ...

approved in Europe to treat the rare disease ADA-SCID, or "bubble boy syndrome." 'Massive challenge'Claire Booth, professor of gene therapy and pediatric immunology at University College London ...

SBMA is also known as Kennedy's disease (KD) named after Dr. William R. Kennedy, who first described the condition. The collaboration aims to identify a small molecule that targets mutant androgen ...

Gael, a patient at St Jude Children's Research Hospital who received treatment, with his mother US scientists say they used HIV to make a gene therapy that cured eight infants of severe combined ...