StitchR makes it possible to deliver larger payloads with current vectors, expanding the list of diseases targetable by gene therapies.
StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.
Researchers unveil the inner mechanisms of the most intricate and complex molecular machine in human biology. Scientists at the Centre (CRG) in Barcelona have developed the first comprehensive ...
This important manuscript provides insights into the competition between Splicing Factor 1 (SF1) and Quaking (QKI) for binding at the ACUAA branch point sequence in a model intron, regulating exon ...
Discover methods for accurate DNA and RNA quantification and learn how to analyze purity and concentration to enhance ...
Make plans to go to this great festival and try some good chicken bog ...
Or sign-in if you have an account. An mRNA vaccine, a cousin to the COVID vaccines that blunted the pandemic, has entered human trials as a treatment for lung cancer. BNT116 is a striking example ...
Large vaccine companies are reluctant to invest too heavily in the emerging technology due to a gap in knowledge, especially compared to mRNA therapeutics. Despite this, those familiar with ...
Lancet Infect Dis 2024 Apr;24(4):351-360). samRNA therapeutics build on standard mRNA vaccines, wherein an mRNA strand encodes a protein presented by a given virus, cancer cell, or other target; this ...