Chemically-engineered recombinant Adeno-Associated Virus: how chemistry improve rAAV liver gene delivery

Characterisation of AAVs in NHPs and rodents using the AskBio Capsid screening (ABCs) platform

Sustained efficacy and safety up to 3.5 years in adults with glycogen storage disease type Ia (GSDIa): longer-term results from a phase 1/2 clinical trial of DTX401, an AAV8-mediated, liver-directed gene therapy

A novel dual AAV approach for intravitreal gene therapy of Stargardt disease

Selection of clinical doses for SBT101, an AAV9-hABCD1 vector for the treatment of adrenomyeloneuropathy

Gene Replacement Therapy for Spastic Paraplegia 47

Optimization of intrathecal delivery of an infused AAV9 vector for delivery of a gene therapy candidate for adrenomyeloneuropathy in non-human primates

Hardwiring tissue-specific AAV transduction in mice through engineered receptor expression

Characterization of engineered AAV capsids in the retina of mice and in two novel human retina ex vivo models

Barcode screening identifies AAV-KP1 as an AAV capsid that efficiently transduces pancreatic islets with minimal off-target consequences following retrograde pancreatic duct injection in non-human primates

Further characterization of a novel AAV vector and expanded selection criteria platform for muscle gene delivery

Delivery of suicide therapies using a precision virotherapy; novel approaches for pancreatic cancer