1996
Gene Therapy in the United States: A Five-Year Status Report
Ross G, Erickson R, Knorr D, Motulsky A, Parkman R, Samulski J, Straus S, Smith B. Gene Therapy in the United States: A Five-Year Status Report. Human Gene Therapy 1996, 7: 1781-1790. PMID: 8886849, DOI: 10.1089/hum.1996.7.14-1781.Peer-Reviewed Original ResearchConceptsInstitutional review boardLocal institutional review boardFDA approvalHuman gene transfer trialsPhase I/IIGene transfer studiesPhase II studyPhase III studyRecruitment of patientsGene therapyYears of ageGene transfer trialsFinal FDA approvalII studyIII studyTotal patientsMedian timeFirst patientAnatomic distributionExperimental therapiesUnexpected toxicitiesNineteen studiesPatientsAutopsy rateAcademic centers
1990
Retroviral mediated gene transfer into bone marrow progenitor cells: use of beta-galactosidase as a selectable marker
Strair R, Towle M, Smith B. Retroviral mediated gene transfer into bone marrow progenitor cells: use of beta-galactosidase as a selectable marker. Nucleic Acids Research 1990, 18: 4759-4762. PMID: 2118622, PMCID: PMC331937, DOI: 10.1093/nar/18.16.4759.Peer-Reviewed Original Research