Self-Inactivating CRISPR-Cas9 System Reduces mHTT With Fewer Off-Target Effects in HD Mice Researchers developed a self-inactivating CRISPR-Cas9 gene-editing approach that reduced mHTT with improved safety in HD mice.

Researchers developed a gene-editing approach that effectively reduced mutant huntingtin (#mHTT) levels and reversed key features of #HuntingtonDisease (HD) in a mouse model. Reported in Science Advances

Read more: https://bit.ly/3NLNDJL

#RareDisease #GeneEditing #MedSky

Chimera Crosses Blood-Brain Barrier to Degrade mHTT Aggregates in Huntington Disease A compound with therapeutic potential in Huntington disease (HD) preferentially degrades mHTT aggregates and improves survival in model mice.

As reported in the @jacs.acspublications.org researchers demonstrated that a proteolysis-targeting chimera (#PROTAC) penetrates the brain to preferentially degrade mutant huntingtin (#mHTT) aggregates in #HuntingtonDisease (HD) mice.

Read here: https://bit.ly/4kTKS5d

#RareDisease #MedSky

Can we identify biomarkers responsive to mHTT lowering?

In this new study, we investigated 💧 #CSF and ☢️ #PET biomarkers that could be used non-invasively to track 📈 the temporal response to #mHTT modulation in mice 🐭.

#openaccess article: 👉 lnkd.in/eqTirauF