Gene editing on center stage

RO Bak, N Gomez-Ospina, MH Porteus - Trends in Genetics, 2018 - cell.com
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 new class of medicines through DNA editing

MH Porteus - New England Journal of Medicine, 2019 - Mass Medical Soc
Clinical Genome Editing Therapeutic editing of the human genome has long been a holy
grail of genetic medicine. Different approaches are founded on a bedrock of basic laboratory …

After the storm—a responsible path for genome editing

GQ Daley, R Lovell-Badge… - New England Journal of …, 2019 - Mass Medical Soc
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 …

The societal opportunities and challenges of genome editing

D Carroll, RA Charo - Genome biology, 2015 - Springer
The genome editing platforms currently in use have revolutionized the field of genetics. At an
accelerating rate, these tools are entering areas with direct impact on human well being …

Advances in genome editing through control of DNA repair pathways

CD Yeh, CD Richardson, JE Corn - Nature cell biology, 2019 - nature.com
Eukaryotic cells deploy overlapping repair pathways to resolve DNA damage.
Advancements in genome editing take advantage of these pathways to produce permanent …

Base editing: advances and therapeutic opportunities

EM Porto, AC Komor, IM Slaymaker… - Nature Reviews Drug …, 2020 - nature.com
Base editing—the introduction of single-nucleotide variants (SNVs) into DNA or RNA in
living cells—is one of the most recent advances in the field of genome editing. As around …

Towards a new era in medicine: therapeutic genome editing

MH Porteus - Genome biology, 2015 - Springer
Genome editing is the process of precisely modifying the nucleotide sequence of the
genome. It has provided a powerful approach to research questions but, with the …

Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects

H Li, Y Yang, W Hong, M Huang, M Wu… - Signal transduction and …, 2020 - nature.com
Based on engineered or bacterial nucleases, the development of genome editing
technologies has opened up the possibility of directly targeting and modifying genomic …

Gene therapy comes of age

CE Dunbar, KA High, JK Joung, DB Kohn, K Ozawa… - Science, 2018 - science.org
BACKGROUND Nearly five decades ago, visionary scientists hypothesized that genetic
modification by exogenous DNA might be an effective treatment for inherited human …

Nuclease‐mediated genome editing: At the front‐line of functional genomics technology

T Sakuma, K Woltjen - Development, growth & differentiation, 2014 - Wiley Online Library
Genome editing with engineered endonucleases is rapidly becoming a staple method in
developmental biology studies. Engineered nucleases permit random or designed genomic …