GENE THERAPY AND SICKLE-CELL ANEMIA

Alaska cousins

Each 12.5% me

Thanks to my friend Phil Montague for tipping me off to this valuable info-bit.

You probably know what sickle- cell anemia is: an excruciatingly painful disease caused by misshapen red blood cells.  They – the blood cells – get this way because of a genetic mistake involving just one letter of the DNA alphabet.  It turns out that NIH has sponsored research leading to a cure – an actual cure – for sickle-cell anemia.  The process is described in a segment of last Sunday’s 60 Minutes TV show (3/10/19), largely through an interview with Dr. Francis Collins, Director of NIH (see https://ljb-quiltcutie.blogspot.com/2017/12/frankenstuff.html).  If you have a TV you should hasten to watch this program, especially if you are still shaky on how gene therapy works.  (And aren’t we all?). 

Several times previously I have declared Dr. Saul Rivkin to be my hero.  Saul lost his wife to OVCA at an early age; he responded by establishing the Marsha Rivkin Center for Ovarian Cancer Research, while raising at least $15 million for its support.  But to Saul I now add Dr. Collins, who headed the official effort to decode DNA, and now serves in a capacity to greatly influence where medical research funds go.  Dr. Collins is a brilliant scientist, an articulate blogger (see NIH web site) – and he even plays a mean guitar!