RT Book, Section
A1 Notarangelo, Luigi D.
A2 Kline, Mark W.
SR Print(0)
ID 1182931700
T1 Gene Therapy for Primary Immune Deficiencies
T2 Rudolph's Pediatrics, 23e
YR 2018
FD 2018
PB McGraw-Hill Education
PP New York, NY
SN 9781259588594
LK accesspediatrics.mhmedical.com/content.aspx?aid=1182931700
RD 2024/04/19
AB Identification  of  the  molecular  basis  of  the  disease  and  development  of  more  effective  strategies  of  gene  transfer  into  hematopoietic  stem  cells  (HSCs)  have  opened  the  way  to  innovative  forms  of  gene  therapy  for  primary  immunodeficiency  (PID).  In  these  approaches,  a  normal  copy  of  the  gene  of  interest  is  introduced  into  the  genome  of  autologous  HSCs  that  are  then  re-infused  into  the  patient.  Gene  therapy  also  holds  the  potential  for  gene  editing,  which  is  actually  the  removal  and  correction  of  an  aberration  in  an  individual  PID-causing  gene.  In  this  chapter,  we  will  review  the  current  status  of  gene  therapy  (including  gene  editing)  for  PIDs  and  discuss  possible  developments  in  the  field.