Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa
Tag: Gene therapy
Adeno-associated virus serotype 2 capsids with proteolytic cuts by trypsin remain intact and potent
Recombinant adeno-associated viral (AAV) vectors have emerged as prominent gene delivery vehicles for gene therapy. In the journey of an AAV vector…
Co-delivery of IL-1Ra and SOX9 via AAV inhibits inflammation and promotes cartilage repair in surgically induced osteoarthritis animal models
Osteoarthritis (OA), a prevalent joint disorder, can lead to disability, with no effective treatment available. Interleukin-1 (IL-1) plays a crucia…
Incomplete elimination of viral genomes is associated with chronic inflammation in nonhuman primate livers after AAV-mediated gene transfer
The liver is a unique organ where immunity can be biased toward ineffective response notably in the context of viral infections. Chronic viral hepa…
A peptide conjugate enables systemic injection of the morpholino inducer and more durable induction of T3H38 ribozyme-controlled AAV transgene in mice
Genetic switches that allow for precise control over transgene expression timing or levels may improve the safety and expand the use of adeno-assoc…
sLZIP functions as a key modulator of bone remodeling by regulating the crosstalk between osteoblasts and osteoclasts
Human small leucine zipper protein (sLZIP) regulates the differentiation of both osteoblasts (OBs) and osteoclasts (OCs). However, the regulatory r…