Over the past decade, in vivo gene replacement therapy has significantly advanced,
resulting in market approval of numerous therapeutics predominantly relying on adeno …

The rate of neurodegenerative disorders (NDDs) is rising rapidly as the world's population
ages. Conditions such as Alzheimer's disease (AD), Parkinson's disease (PD), and …

Familial dysautonomia (FD) is a currently untreatable, neurodegenerative disease caused
by a splicing mutation (c. 2204+ 6T> C) that causes skipping of exon 20 of the elongator …

Lentiviral vectors are attractive delivery vehicles for cystic fibrosis gene therapy owing to
their low immunogenicity and ability to integrate into the host cell genome, thereby …

In this study, the author compared the performance of two allometric scaling approaches and
body-weight-based dose conversion approach for first-in-patient (FIP) dose prediction for …

To facilitate model-informed drug development (MIDD) of adeno-associated virus (AAV)
therapy, here we have developed a physiologically based pharmacokinetic (PBPK) model …

Poly (β-amino ester)(PBAE) nanoparticles (NPs) show great promise for nonviral gene
delivery. Recent studies suggest branched PBAEs (BPBAEs) offer advantages over linear …

Nonalcoholic Steatohepatitis represents a severe disease subtype of nonalcoholic fatty liver
disease which is thought to be highly associated with systemic metabolic abnormalities. It is …

Mucopolysaccharidoses type IIIB is a rare genetic disorder caused by mutations in the gene
that encodes for N-acetyl-alpha-glucosaminidase. This results in the aggregation of heparan …

This study examines inhibiting galectin 1 (Gal1) as a treatment option for hepatocellular
carcinoma (HCC). Gal1 has immunosuppressive and cancer-promoting roles. Our data …