BACKGROUND Nearly five decades ago, visionary scientists hypothesized that genetic
modification by exogenous DNA might be an effective treatment for inherited human …

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 …

We present an overview of clinical trials involving gene editing using clustered interspaced
short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription …

Genome editing has the potential to treat an extensive range of incurable monogenic and
complex diseases. In particular, advances in sequence-specific nuclease technologies have …

▪ Abstract Broadly defined, the concept of gene therapy involves the transfer of genetic
material into a cell, tissue, or whole organ, with the goal of curing a disease or at least …

Despite the ups and downs in the field over three decades, the science of gene therapy has
continued to advance and provide enduring treatments for increasing number of diseases …

Although high upfront costs for the high value of gene therapy have resulted in concerns
about sufficient reimbursement to allow patient access to these therapies, the significant …

In Part I of this Review, we introduced recent advances in gene delivery technologies and
explained how they have powered some of the current human gene therapy applications. In …

Smithies et al.(1985) and Jasin and colleagues (1994) provided proof of concept that
homologous recombination (HR) could be applied to the treatment of human disease and …

A Path Forward for Genome Editing Though the first application of embryo editing was
deeply flawed, not least because it didn't address an unmet clinical need, and has prompted …