TY - JOUR
T1 - Antiviral and anticancer ribozymes
AU - Poeschla, E.
AU - Wong-Staal, F.
PY - 1994
Y1 - 1994
N2 - Molecular biologists are beginning to apply the site-specific RNase properties of ribozymes to gene therapy for HIV infection and cancer. Requirements for substrate recognition and cleavage are being finely mapped. Methods of augmenting intracellular cleavage and of dissecting observed discrepancies between in vitro and cellular activity are being explored. Antiviral efficacy against HIV type 1 has been seen in tissue culture for both hammerhead and hairpin ribozymes, and a phase I clinical trial for ex vivo T-cell gene therapy is planned. Oncogene transcripts, such as the bcr/abl fusion messenger RNA and ras, have also been targeted with ribozymes.
AB - Molecular biologists are beginning to apply the site-specific RNase properties of ribozymes to gene therapy for HIV infection and cancer. Requirements for substrate recognition and cleavage are being finely mapped. Methods of augmenting intracellular cleavage and of dissecting observed discrepancies between in vitro and cellular activity are being explored. Antiviral efficacy against HIV type 1 has been seen in tissue culture for both hammerhead and hairpin ribozymes, and a phase I clinical trial for ex vivo T-cell gene therapy is planned. Oncogene transcripts, such as the bcr/abl fusion messenger RNA and ras, have also been targeted with ribozymes.
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U2 - 10.1097/00001622-199411000-00012
DO - 10.1097/00001622-199411000-00012
M3 - Review article
C2 - 7827172
AN - SCOPUS:0028143351
SN - 1040-8746
VL - 6
SP - 601
EP - 606
JO - Current Opinion in Oncology
JF - Current Opinion in Oncology
IS - 6
ER -