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Genetic vectors are vehicles for delivering foreign DNA into recipient cells. Vectors can replicate autonomously and typically include features to facilitate the manipulation of DNA as well as a genetic marker for their selective recognition. The most common vectors are DNA plasmids, viruses and artificial chromosomes.
Bradford, Zhang and colleagues generate a mouse model harboring a mutation that impacts PKP2 splicing and show that one-time administration of AAV-PKP2 in neonatal mice could restore PKP2 and prevent the onset of key pathological features of arrhythmogenic right ventricular cardiomyopathy, and one-time administration in adult mice could rescue the phenotype and prevent sudden death up to 4 months post treatment.
A study in Nature Biotechnology reports a platform that combines lentivirus capabilities with antibody recognition for targeted cell delivery and genome editing.
Arrhythmogenic cardiomyopathy is a major cause of sudden death among young people. Three studies show that gene therapy to restore the desmosomal protein PKP2 holds promise in improving the prognosis of affected individuals.
The COVID-19 pandemic brought mRNA vaccines to market in a short period, pointing the entire drug development field in the direction of mRNA treatment.
Ultrasensitive sequential fluorescence in situ hybridization (USeqFISH) enables multiplexed detection of the expression of endogenous and exogenous genes delivered by adeno-associated virus (AAV) vectors in intact tissue. USeqFISH provides a spatial map of AAV tropism with high throughput and resolution.
