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

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

Genome editing technologies, particularly those based on zinc-finger nucleases (ZFNs),
transcription activator-like effector nucleases (TALENs), and CRISPR (clustered regularly …

Gene-editing pipeline takes off. - Document - Gale OneFile: Health and Medicine Use this link
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Gene therapy has historically been defined as the addition of new genes to human cells.
However, the recent advent of genome-editing technologies has enabled a new paradigm in …

The clinical application of ex vivo gene edited cell therapies first began a decade ago with
zinc finger nuclease editing of autologous CD4+ T-cells. Editing aimed to disrupt expression …

Recent progress in developing programmable nucleases, such as zinc-finger nucleases,
transcription activator–like effector nucleases (TALENs) and clustered regularly interspaced …

Hematopoietic stem and progenitor cells (HSPCs) have great therapeutic potential because
of their ability to both self-renew and differentiate. It has been proposed that, given their …

Based on engineered or bacterial nucleases, the development of genome editing
technologies has opened up the possibility of directly targeting and modifying genomic …

The ability to precisely modify human genes has been made possible by the development of
tools such as meganucleases, zinc finger nucleases, TALENs, and CRISPR/Cas. These now …