Exploring the therapeutic potential of modulating nonsense-mediated mRNA decay
M McMahon, LE Maquat - RNA, 2024 - rnajournal.cshlp.org
Discovered more than four decades ago, nonsense-mediated mRNA decay (NMD) plays a
fundamental role in the regulation of gene expression and is a major contributor to …
fundamental role in the regulation of gene expression and is a major contributor to …
[HTML][HTML] Conventional and Tropism-Modified High-Capacity Adenoviral Vectors Exhibit Similar Transduction Profiles in Human iPSC-Derived Retinal Organoids
A McDonald, C Gallego, C Andriessen… - International …, 2024 - pmc.ncbi.nlm.nih.gov
Viral vector delivery of gene therapy represents a promising approach for the treatment of
numerous retinal diseases. Adeno-associated viral vectors (AAV) constitute the primary …
numerous retinal diseases. Adeno-associated viral vectors (AAV) constitute the primary …
Novel Gene Therapy Approach Uses 'Stitched'Together mRNAs to Deliver Large Therapeutic Therapies for Muscular Dystrophies
J Talan - Neurology Today, 2025 - journals.lww.com
Researchers molecularly stitched together two separate messenger RNAs to deliver and
restore each therapeutic protein at or above wild-type levels and to almost all muscle fibers …
restore each therapeutic protein at or above wild-type levels and to almost all muscle fibers …
StitchR platform joins up gene therapy payloads
K Kingwell - Nature reviews. Drug discovery - researchgate.net
Adeno-associated virus (AAV) gene therapy holds considerable promise for treating
diseases caused by genetic defects or deficiencies, but the packaging capacity of AAV …
diseases caused by genetic defects or deficiencies, but the packaging capacity of AAV …