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Gene Editing Basics

Therapeutic Gene Editing: An ASGCT White Paper

Gene editing is a process that repairs or changes a gene. Medical researchers are exploring ways to use gene editing to treat or prevent human diseases. This report provides a foundation for understanding the science of gene editing, its potential therapeutic applications and its current U.S. regulatory framework.

Click HERE to view the White Paper.

ASGCT conducted a briefing before the U.S. Senate Health, Education, Labor, and Pension (HELP) Committee and met with representatives of the White House Office of Science and Technology Policy on November 18, 2016.  The purpose of these meetings was to provide educational information on therapeutic gene editing in humans, coupled with the ASGCT White Paper. 

The National Academy of Sciences and the National Academy of Medicine are presently preparing a consensus report on gene editing, to be released in early 2017, the scope and influence of which is expected to be broad.  ASGCT is extending its scientific expertise to provide ample background information on gene editing technology and its therapeutic approaches to allow key decision-makers in Congress and the Administration to better digest the content of the highly anticipated NAS consensus report.

View the presentation to the briefing HERE.