RT Book, Section
A1 Gladwin, Mark T.
A1 Kato, Gregory J.
A1 Novelli, Enrico M.
SR Print(0)
ID 1179337689
T1 Foreword
T2 Sickle Cell Disease
YR 2021
FD 2021
PB McGraw-Hill Education
PP New York, NY
SN 9781260458596
LK hemonc.mhmedical.com/content.aspx?aid=1179337689
RD 2024/04/19
AB The  publication  of  Sickle  Cell  Disease  couldn't  be  timelier.  Following  Herrick's  initial  description  of  the  clinical  manifestations  in  1910  and  the  discovery  of  sickle  hemoglobin  by  Pauling  and  Itano  nearly  50  years  later,  there  has  been  substantial,  albeit  slow,  progress  in  working  out  the  molecular  and  cellular  pathogenesis  of  the  disease  as  well  as  the  development  of  safe  and  effective  therapies.  For  many  decades  funding  both  from  the  National  Institutes  of  Health  and  the  pharmaceutical  industry  for  sickle  cell  disease  has  been  disproportionately  low,  considering  the  importance  of  this  disease,  both  in  prevalence  and  severity.  During  this  period  of  "slow  growth,"  enticing  enough  young  well-trained  lab  and  clinical  investigators  to  work  on  this  disease  was  a  challenge.  Happily,  during  the  past  several  years,  sickle  cell  research  has  gained  a  robust  lease  on  life.  The  impressive  up-swing  in  scientific  and  clinical  abstracts  and  presentations  at  recent  national  meetings  bears  this  out.  Importantly,  Pharma  is  now  making  a  major  commitment,  with  the  development  of  an  impressive  array  of  novel  drugs  that  target  critical  sites  in  the  pathogenesis  pathway.  This  well-spring  of  enthusiasm  has  culminated  in  the  realization,  during  the  past  year,  that  gene  therapy  is  now  in  hand,  with  realistic  prospects  for  disease  amelioration  and  possible  cure.  With  the  expanding  cadre  of  multidisciplinary  scientists  and  clinicians  entering  research  on  sickle  cell  disease,  this  all-encompassing  book  will  be  a  valuable  resource.