a friend pointed out that just because locus are of small effect, one might still be able to target that locus with a drug and case a huge phenotypic change. so the polygenic part of the newsletter may be on the pessimistic side.
The PI for that trial is the man who was PI for the development of Zolgensma (onasemnogene abeparvovec-xioi) which targets the genetic root cause of spinal muscular atrophy in infants by replacing the function of the missing or nonworking SMN1 gene with a new, working copy of a human SMN gene. https://www.zolgensma.com/how-zolgensma-works.
We are living in the 21st Century.
We should also give some credit to genetic technology for the creation of the 2 new vaccines against Coronavirus. Less than a year after the virus was identified and sequenced. It is an awesome accomplishment. Further, the underlying mRNA technology could be extended to other diseases, including cancers.
a friend pointed out that just because locus are of small effect, one might still be able to target that locus with a drug and case a huge phenotypic change. so the polygenic part of the newsletter may be on the pessimistic side.
here is the explanation https://www.gnxp.com/blog/2010/02/small-genetic-effects-do-not-preclude.php
There is an ongoing clinical trial of a treatment for Duchenne Muscular Dystrophy by Systemic Gene Delivery https://www.clinicaltrials.gov/ct2/show/NCT03375164
The PI for that trial is the man who was PI for the development of Zolgensma (onasemnogene abeparvovec-xioi) which targets the genetic root cause of spinal muscular atrophy in infants by replacing the function of the missing or nonworking SMN1 gene with a new, working copy of a human SMN gene. https://www.zolgensma.com/how-zolgensma-works.
We are living in the 21st Century.
We should also give some credit to genetic technology for the creation of the 2 new vaccines against Coronavirus. Less than a year after the virus was identified and sequenced. It is an awesome accomplishment. Further, the underlying mRNA technology could be extended to other diseases, including cancers.