Clustered regularly interspaced short palindromic repeats (CRISPR) system provides
adaptive immunity against plasmids and phages in prokaryotes. This system inspires the …

Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …

Background Moderate-to-severe hemophilia B is treated with lifelong, continuous
coagulation factor IX replacement to prevent bleeding. Gene therapy for hemophilia B aims …

Background Valoctocogene roxaparvovec delivers a B-domain–deleted factor VIII coding
sequence with an adeno-associated virus vector to prevent bleeding in persons with severe …

Abstract Background Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-
associated virus 5 (AAV5)–based gene-therapy vector containing a coagulation factor VIII …

Abstract Background Patients with the Crigler–Najjar syndrome lack the enzyme uridine
diphosphoglucuronate glucuronosyltransferase 1A1 (UGT1A1), the absence of which leads …

Background The goal of gene therapy for patients with hemophilia A is to safely impart long-
term stable factor VIII expression that predictably ameliorates bleeding with the use of the …

Nine dogs with hemophilia A were treated with adeno-associated viral (AAV) gene therapy
and followed for up to 10 years. Administration of AAV8 or AAV9 vectors expressing canine …

Nanoparticle delivery to solid tumours over the past ten years has stagnated at a median of
0.7% of the injected dose. Varying nanoparticle designs and strategies have yielded only …

Adeno-associated virus (AAV) vector-mediated gene delivery was recently approved for the
treatment of inherited blindness and spinal muscular atrophy, and long-term therapeutic …