Gene therapy and bone marrow transplant alleviate ultra-rare genetic disease symptoms
In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...
ophthalmologytimes3d
Viralgen and Axovia Therapeutics partner to manufacture gene therapy for Bardet-Biedl Syndrome
The companies will work together by using the codon-optimized BBS1 AAV9 vector to minimize the vision loss caused by the ...
Medpage Today on MSN4d
Gene Therapy Stabilizes Rare Chronic HPV Disease
For recurrent respiratory papillomatosis (RRP), novel gene therapy to jump-start immunity to the human papillomavirus (HPV) ...
PharmiWeb4d
Reithera and Sillajen Partnership Announced
ReiThera is proud to announce a new partnership with SillaJen through a comprehensive Master Service Agreement (MSA), which establishes ReiThera as the manufacturing partner for SillaJen’s innovative ...
pharmaphorum2d
FDA approves Adstiladrin as first gene therapy for NMIBC
Ferring Pharma’s Adstiladrin is a non-replicating (cannot multiply in human cells) adenoviral vector-based gene therapy ... NICE recommended routine use of Bristol-Myers Squibb’s Opdivo ...
Next-generation gene-editing tool offers enhanced capabilities for cancer and medical research
Australian cancer researchers are the first to establish a next-generation gene-editing tool for modeling and interrogating ...
Nature3d
Gene therapy articles from across Nature Portfolio
An in vivo base editing approach targeting the PRNP gene led to a 52% increase in the lifespan of mouse models inoculated with the most common sporadic and genetic types of human pathogenic prion ...
The Chronicle21h
Duke professor-led biotech company raises $175 million to advance epigenome editing therapy clinical trials
The company will use the funding to advance clinical trials for Tune-401, the epigenetic silencing drug for treating chronic ...
Escaping the endosome: BEND lipids improve LNP mRNA delivery and gene editing
Every time a shuttle docks with the International Space Station (ISS), a delicate dance unfolds between the shuttle's docking ...
News Medical on MSN3d
BEND lipids revolutionize mRNA delivery and gene-editing
Every time a shuttle docks with the International Space Station (ISS), a delicate dance unfolds between the shuttle's docking system and its counterpart on the station.
ascopubs.org5d
Use of gene expression profiling to examine association between MUC1 and claudin 18 in gastrointestinal cancers: Implications for targeted therapies.
Short interval circulating tumor DNA (ctDNA) kinetics as a predictor of tumor response in patients with gastrointestinal (GI) cancer receiving immune checkpoint inhibitor (ICI)-based treatment. This ...