TY - JOUR
T1 - Gene therapy with viral vectors
AU - Kootstra, Neeltje A.
AU - Verma, Inder M.
PY - 2003
Y1 - 2003
N2 - A key factor in the success of gene therapy is the development of gene delivery systems that are capable of efficient gene transfer in a broad variety of tissues, without causing any pathogenic effect. Currently, viral vectors based on many different viruses have been developed, and their performance and pathogenicity has been evaluated in animal models. The results of these studies form the basis for the first clinical trials for correcting genetic disorders using retroviral, adenoviral, and adeno-associated viral vectors. Even though the results of these trials are encouraging, vector development is still required to improve and refine future treatment of hereditary disorders
AB - A key factor in the success of gene therapy is the development of gene delivery systems that are capable of efficient gene transfer in a broad variety of tissues, without causing any pathogenic effect. Currently, viral vectors based on many different viruses have been developed, and their performance and pathogenicity has been evaluated in animal models. The results of these studies form the basis for the first clinical trials for correcting genetic disorders using retroviral, adenoviral, and adeno-associated viral vectors. Even though the results of these trials are encouraging, vector development is still required to improve and refine future treatment of hereditary disorders
U2 - https://doi.org/10.1146/annurev.pharmtox.43.100901.140257
DO - https://doi.org/10.1146/annurev.pharmtox.43.100901.140257
M3 - Review article
C2 - 12359866
SN - 0362-1642
VL - 43
SP - 413
EP - 439
JO - Annual review of pharmacology and toxicology
JF - Annual review of pharmacology and toxicology
ER -