RNA-based gene therapy requires therapeutic RNA to function inside target cells without
eliciting unwanted immune responses. RNA can be ferried into cells using non-viral drug …

Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

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

Gene transfer using adeno-associated viral (AAV) vectors has made tremendous progress in
the last decade and has achieved cures of debilitating diseases such as hemophilia A and …

CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however,
these drugs must enter the desired cell without eliciting an unwanted immune response, so …

Throughout its 40-year history, the field of gene therapy has been marked by many
transitions. It has seen great strides in combating human disease, has given hope to patients …

Gene therapy is at an inflection point. Recent successes in genetic medicine have paved the
path for a broader second wave of therapies and laid the foundation for next-generation …

While there has been substantial progress and notable achievements in the use of adeno-
associated virus (AAV) gene therapy vectors for treatment of rare diseases, setbacks related …

In vivo gene therapy is rapidly emerging as a new therapeutic paradigm for monogenic
disorders. For almost three decades, hemophilia A (HA) and hemophilia B (HB) have served …

Recombinant adeno-associated virus (AAV) is the leading vector for gene therapy in the
retina. As non-pathogenic, non-integrating, replication deficient vector, the recombinant virus …