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