Question

what are the International Commission on the Clinical use of Heritble Human Genome Editing proposed set of strict preclinical and clinical criteria that would need to be fulfilled before considering heritable human genome editing as safe and effective.
The answer must have authority

Answer 1

The International Commission on the Clinical use of Heritable Human Genome Editing, convened by the U.S. National Academy of Medicine, the U.S. National Academy of Sciences, and the UK's Royal Society, proposed a set of strict preclinical and clinical criteria in their 2020 report "Heritable Human Genome Editing" in order to ensure the safety and efficacy of heritable human genome editing.

These criteria include:

1. Confirming the presence of a pathogenic variant in the gene to be edited and establishing that editing can correct the variant without introducing unintended changes.

2. Demonstrating the safety and efficacy of the editing technology in relevant preclinical models, including non-human primates or other appropriate model organisms.

3. Conducting rigorous preclinical studies to assess potential off-target effects of the editing technology and ensure that the edited embryos are healthy and viable.

4. Developing a clear protocol for embryo selection and transfer that minimizes the risks of mosaicism and off-target effects.

5. Demonstrating long-term follow-up of edited individuals to monitor for any potential adverse effects or unintended consequences of the editing.

By adhering to these criteria, the Commission believes that researchers and clinicians can responsibly pursue heritable human genome editing in a safe and effective manner.