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 …

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 …

Prime editing is a highly versatile CRISPR-based genome editing technology that works
without DNA double-strand break formation. Despite rapid technological advances, in vivo …

There has been a resurgence in gene therapy efforts that is partly fueled by the identification
and understanding of new gene delivery vectors. Adeno-associated virus (AAV) is a non …

In recent years, the number of clinical trials in which adeno-associated virus (AAV) vectors
have been used for in vivo gene transfer has steadily increased. The excellent safety profile …

Gene transfer has long been a compelling yet elusive therapeutic modality. First mainly
considered for rare inherited disorders, gene therapy may open treatment opportunities for …

Duchenne muscular dystrophy (DMD) is an X-linked, muscle wasting disease that affects 1
in 5000 males. Affected individuals become wheelchair bound by the age of twelve and …

Both observational and perturbational technologies are essential for advancing the
understanding of brain function and dysfunction. But while observational techniques have …

The successful use of clustered regularly interspaced short palindromic repeat
(CRISPR)/Cas9-based gene editing for therapeutics requires efficient in vivo delivery of the …

Several attempts have been made to discover the ideal vector for gene therapy in central
nervous system (CNS). Adeno-associated viruses (AAVs) are currently the preferred vehicle …