{"title":"新的基于重组腺相关病毒(rAAV)的治疗方法进入关键的2期临床试验","authors":"C. Mueller, S. Kaushal, M. Humphries, T. Flotte","doi":"10.1142/S1568558611000295","DOIUrl":null,"url":null,"abstract":"This brief review summarizes the design of two phase 2 clinical trials of recombinant adeno-associated virus (rAAV)-based vector that are currently underway. These include a trial of a rAAV2-RPE65 vector for patients with Leber congenital amaurosis and a trial of a rAAV1-alpha-1-antitrypsin(AAT) vector delivered intramuscularly in patients with genetic emphysema due to AAT deficiency. In both cases, the current phase 2 trials were preceded by phase 1 trials indicating a good safety profile and persistent transgene expression.","PeriodicalId":93646,"journal":{"name":"Gene therapy and regulation","volume":null,"pages":null},"PeriodicalIF":0.0000,"publicationDate":"2011-11-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1142/S1568558611000295","citationCount":"0","resultStr":"{\"title\":\"NEW RECOMBINANT ADENO-ASSOCIATED VIRUS (rAAV)-BASED THERAPEUTICS REACH PIVOTAL PHASE 2 CLINICAL TRIALS\",\"authors\":\"C. Mueller, S. Kaushal, M. Humphries, T. Flotte\",\"doi\":\"10.1142/S1568558611000295\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"This brief review summarizes the design of two phase 2 clinical trials of recombinant adeno-associated virus (rAAV)-based vector that are currently underway. These include a trial of a rAAV2-RPE65 vector for patients with Leber congenital amaurosis and a trial of a rAAV1-alpha-1-antitrypsin(AAT) vector delivered intramuscularly in patients with genetic emphysema due to AAT deficiency. In both cases, the current phase 2 trials were preceded by phase 1 trials indicating a good safety profile and persistent transgene expression.\",\"PeriodicalId\":93646,\"journal\":{\"name\":\"Gene therapy and regulation\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2011-11-20\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://sci-hub-pdf.com/10.1142/S1568558611000295\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Gene therapy and regulation\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1142/S1568558611000295\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Gene therapy and regulation","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1142/S1568558611000295","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
This brief review summarizes the design of two phase 2 clinical trials of recombinant adeno-associated virus (rAAV)-based vector that are currently underway. These include a trial of a rAAV2-RPE65 vector for patients with Leber congenital amaurosis and a trial of a rAAV1-alpha-1-antitrypsin(AAT) vector delivered intramuscularly in patients with genetic emphysema due to AAT deficiency. In both cases, the current phase 2 trials were preceded by phase 1 trials indicating a good safety profile and persistent transgene expression.
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