RT Book, Section
A1 McCarthy, Jeanette J.
A1 Mendelsohn, Bryce A.
SR Print(0)
ID 1134735617
T1 Horizons in Genomic Medicine
T2 Precision Medicine: A Guide to Genomics in Clinical Practice
YR 2016
FD 2016
PB McGraw-Hill Education
PP New York, NY
SN 9781259644139
LK accessmedicine.mhmedical.com/content.aspx?aid=1134735617
RD 2024/04/20
AB The  possibility  of  gene  editing  (actually  physically  fixing  a  disease-causing  mutation)  in  an  embryo  or  human  tissue  is  quickly  becoming  a  reality.  Several  different  technologies  permit  gene  editing,  but  currently  the  most  promising  is  based  on  CRISPR/Cas9.  The  Cas9  enzyme  is  part  of  a  rudimentary  bacterial  immune  system  that  binds  to  a  guide  RNA,  finds  the  complementary  DNA  sequence,  and  cuts  it.  Bacteria  use  the  CRISPR  system  to  defend  themselves  against  invading  viruses,  but  the  same  technology  can  be  harnessed  to  make  a  cut  almost  anywhere  in  the  human  genome.  Additional  DNA  can  then  be  added  to  replace  the  DNA  around  the  cut  site.  Any  alteration  in  an  embryo  or  in  a  germ  cell  (predecessor  to  an  egg  or  sperm)  would  be  passed  on  indefinitely  in  future  generations.  This  technology  is  not  yet  highly  efficient,  but  progress  has  been  stunning  and  major  advancements  are  inevitable.