as declared in However, since gene editing methods have the potential to permanently modify a gene, we sought to determine if similar vectors could achieve a body-wide gene repair in muscle tissues.
We became interested in gene editing as a potential alternative to gene replacement.
Our major focus has been on a therapy to replace the defective gene (called the dystrophin gene) in the muscle by delivering a synthetic replacement.
This formed the basis for our gene replacement methods using micro-dystrophins.
We found that delivery of the CRISPR/Cas9 components using AAV did indeed lead to gene editing in muscles body-wide, although to date the efficiency is significantly lower than our previous gene replacement approach.We also tested four different ways to edit the dystrophin gene.
As it stated in
Human gene editing ‘could be allowed to tackle diseases’
They add that gene editing for enhancement should not be allowed “at this time” – but do not rule it out completely.
Many critics have argued that powerful new gene editing techniques should never be used to alter inherited DNA.
Modifications to inherited human DNA could be permitted in future in order to treat or prevent diseases that may be passed onto future generations, two major US scientific institutions have said.
Future use of germline gene editing to treat or prevent disease and disability is a “realistic possibility that deserves serious consideration”, the report says.
Human Genetics Alert calls for an international campaign against the creation of GM babies and human cloning.”
As it stated in
Proceed with caution with gene editing
“Although heritable germline genome editing trials must be approached with caution, the committee said caution does not mean prohibition,” said Rossant.
More contentious is the notion of “germline” editing, in which the sperm, egg or early embryo has its DNA altered.
For instance, genome editing could be used to treat such genetic diseases as sickle cell anemia and thalassemia, Rossant said.
Many view germline editing as crossing an “ethically inviolable” line, the report said.
“That’s called somatic genome editing.
about the details read more from here
This content may collect you by Emily Henry