//
sign in
Post
by @danabra.mov
PostEmbed
by @danabra.mov
Record
by @jimpick.com
Record
by @atsui.org
+ new component
Post
Hot off the press from the RTI: Systemic dual-gene therapy reverses biochemical intoxication in the central metabolic compartment of Bckdha-/- mice pubmed.ncbi.nlm.nih.gov/42136029/?ut... #RNA #RNATherapeutics
27d
Branched-chain 2-ketoacid dehydrogenase (BCKDH) deficiency (maple syrup urine disease; MSUD) causes lethal encephalopathy by disrupting cerebral metabolism, a process imperfectly reflected by circulating...
Systemic dual-gene therapy reverses biochemical intoxication in the central metabolic compartment of Bckdha-/- mice - PubMed
pubmed.ncbi.nlm.nih.gov
RNA Therapeutics Institute