TY - JOUR
T1 - Genetic therapy
T2 - Past, present, and future
AU - Flotte, T. R.
AU - Ferkol, T. W.
N1 - Funding Information:
The authors are supported in part by grants from the CF Foundation and the National Institutes of Health HL51811 (TRF), DK48996 (TWF), and DK27691 (TWF). Dr. Ferkol is also a co-founder of Copernicus Gene Systems, 11100 Cedar Avenue, Cleveland, Ohio.
PY - 1997
Y1 - 1997
N2 - The early stages of genetic therapy present challenges for clinicians and basic scientists. Clinicians must become familiar with new terminology and concepts, and must keep a perspective on the new field in the face of inflated claims and high-profile failures. Basic scientists must continually return to disease models and to patients to determine what are the proper safety issues and relevant efficacy questions for specific diseases and vector systems. And in an era of instant information, all concerned parties must be careful about how progress is communicated to colleagues, patients, and the lay public.
AB - The early stages of genetic therapy present challenges for clinicians and basic scientists. Clinicians must become familiar with new terminology and concepts, and must keep a perspective on the new field in the face of inflated claims and high-profile failures. Basic scientists must continually return to disease models and to patients to determine what are the proper safety issues and relevant efficacy questions for specific diseases and vector systems. And in an era of instant information, all concerned parties must be careful about how progress is communicated to colleagues, patients, and the lay public.
UR - http://www.scopus.com/inward/record.url?scp=0031041468&partnerID=8YFLogxK
U2 - 10.1016/S0031-3955(05)70468-5
DO - 10.1016/S0031-3955(05)70468-5
M3 - Article
C2 - 9057789
AN - SCOPUS:0031041468
SN - 0031-3955
VL - 44
SP - 153
EP - 178
JO - Pediatric Clinics of North America
JF - Pediatric Clinics of North America
IS - 1
ER -