Gene therapy for X-linked myotubular myopathy with AT132 (rAAV8-Des-hMTM1): preliminary results from the ASPIRO phase-1/2 study

著录项

• 来源
  《Human gene therapy》 |2018年第12期|共1页
• 作者

  Kuntz N.; Shieh P. B.; Smith B.; Bonnemann C. G.; Dowling J. J.; Lawlor M. W.; Mavilio F.; Mueller-Felber W.; Noursalehi M.; Rico S.; Servais L.; Prasad S.;

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• 作者单位

  Ann &

  Robert H Lurie Childrens Hosp Chicago IL USA;

  Univ Calif Los Angeles Los Angeles CA USA;

  Univ Florida Gainesville FL USA;

  NIH Porter Neurosci Res Ctr Bldg 10 Bethesda MD 20892 USA;

  Hosp Sick Children Toronto ON Canada;

  Med Coll Wisconsin Milwaukee WI 53226 USA;

  Audentes Therapeut San Francisco CA USA;

  Klinikum Univ Munchen Munich Germany;

  Audentes Therapeut San Francisco CA USA;

  Audentes Therapeut San Francisco CA USA;

  Hop Armand Trousseau I Mot Paris France;

  Audentes Therapeut San Francisco CA USA;

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• 原文格式 PDF
• 正文语种 eng
• 中图分类 治疗学;
• 关键词