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

Expand full comment

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.

Expand full comment