Gene therapy [NEW TOPIC PAGE]

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Last Posted: Oct 06, 2017

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• Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.  
  Eichler Florian et al. The New England journal of medicine 2017 Oct
• In a First, Gene Therapy Halts a Fatal Brain Disease  
  G Kolata, NY Times, Oct 5, 2017
• Gene Therapy for Atrial Fibrillation in Heart Failure.  
  Arora R et al. Clinical pharmacology and therapeutics 2017 Aug 102(2) 200-202
• Gene Therapy Helps Treat Boy's Rare Immune Disorder  
  Dana Farber Inst, Sep 2017
• Gene Therapy Helps Treat Boy’s Rare Immune Disorder  
  Dana Farber Inst, Sep 2017
• Gene Therapy and September Scenes  
  R Lewis, PLOS Blogs, Sep 2017
• FDA Approval of Tisagenlecleucel Promise and Complexities of a $475 000 Cancer Drug  
  PB Bach et al, JAMA, Sep 20, 2017
• Gene Therapy and September Scenes  
  R Lewis, PLOS Blogs, Sep 2017
• New Gene-Therapy Treatments Will Carry Whopping Price Tags  
  G Kolata, NY Times, Sep 11, 2017
• Progress toward Gene Therapy for Duchenne Muscular Dystrophy.  
  Chamberlain Joel R et al. Molecular therapy : the journal of the American Society of Gene Therapy 2017 May 25(5) 1125-1131
• F.D.A. Approves First Gene-Altering Leukemia Treatment, Costing $475,000  
  D Grady, NY Times, August 30, 2017
• FDA Approves First Gene Therapy Treatment For Cancer  
  R Stein, NPR, August 30, 2017
• Deriving genomic diagnoses without revealing patient genomes.  
  Jagadeesh Karthik A et al. Science (New York, N.Y.) 2017 Aug 357(6352) 692-695
• Doctors Plan Bold Test of Gene Therapy on Boys with Muscular Dystrophy  
  A Regalado, MIT Tech Review, August 17, 2017
• Human genomics projects and precision medicine.  
  Carrasco-Ramiro F et al. Gene therapy 2017 Aug
• The ethical framework for performing research with rare inherited neurometabolic disease patients.  
  Giannuzzi Viviana et al. European journal of pediatrics 2017 Mar 176(3) 395-405

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