Genes contain the essential building instructions for life, guiding cells on which amino acids to assemble in what sequence to produce specific proteins. The human genome codes for about 20,000 such ...
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.
An international research team has uncovered a new mechanism crucial to the production of cellular proteins. When this ...
An international research team has uncovered a new mechanism crucial to the production of cellular proteins. When this ...
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 ...
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