GENE THERAPY ▲ Public Health Genomics Knowledge Base (v1.2)

Public Health Genomics Knowledge Base (v1.2)

Last Posted: Nov-10-2016 11AM

 Gene Therapycollapsed collapsed

Snapshots of Life: Lighting up the Promise of Retinal Gene Therapy
Francis Collins, NIH Director, November 10, 2016

Gene therapy for blistering skin disease appears to enhance healing in clinical trial
Stanford Medicine, November 1, 2016

Gene therapy shows promise for treating Niemann-Pick disease type C1
NIH, October 26, 2016

More information on this topic

Last Updated: Nov 10, 2016

• Snapshots of Life: Lighting up the Promise of Retinal Gene Therapy
  Francis Collins, NIH Director, November 10, 2016
• Gene therapy for blistering skin disease appears to enhance healing in clinical trial
  Stanford Medicine, November 1, 2016
• Regulatory Oversight of Cell and Gene Therapy Products in Canada.
  Ridgway Anthony et al. Advances in experimental medicine and biology 2015 49-71
• Gene therapy shows promise for treating Niemann-Pick disease type C1
  NIH, October 26, 2016
• Making gene therapy available at the point of care
  J Harris, PHG Foundation, October 24, 2016
• CLINICAL PROGRESS IN INHERITED RETINAL DEGENERATIONS: GENE THERAPY CLINICAL TRIALS AND ADVANCES IN GENETIC SEQUENCING.
  Hafler Brian P, et al. Retina (Philadelphia, Pa.) 2016 10
• Gene therapy: A new chapter
  A King, Nature Outlook, September 2016
• Gene editing meets gene therapy 
  Genomic Education Program, UK, September 2016
• Gene therapy for sickle cell disease passes key preclinical test
  Harvard Gazettte, September 2016
• Advances in gene therapy for muscular dystrophies.
  Abdul-Razak Hayder et al. F1000Research 2016
• Gene therapy for sickle cell moves closer as scientists clear unexpected obstacle
  StatNews, September 7, 2016
• Gene therapy 2.0: Will CRISPR make expensive treatment accessible to all?
  D Warmflash, Genetic Literacy Project, August 16, 2016
• An innovative strategy for the molecular diagnosis of Usher syndrome identifies causal biallelic mutations in 93% of European patients.
  Bonnet Crystel, et al. European journal of human genetics : EJHG 2016 7
• Safety and Efficacy in Advanced Solid Tumors of Docetaxel in Combination with a Targeted Nanocomplex Carrying the p53 Gene: A Phase Ib Study.
  Pirollo Kathleen F, et al. Molecular therapy : the journal of the American Society of Gene Therapy 2016 6
• Tumor- and neoantigen-reactive T-cell receptors can be identified based on their frequency in fresh tumor.
  Pasetto Anna, et al. Cancer immunology research 2016 6

• Human (46)
• Pathogen (0)

• Human (98)
• Pathogen (0)

• Human (6)
• Pathogen (0)

• Guidelines (1)
• Tier Table (0)
• Synthesis (1)

• Huamn (1)
• Pathogen (0)

Relevant Resources

• Genetics and Genomics Competencies Center (G2C2)
• NIH Genetic Testing Registry
• NIH MedGen Database
• Online Mendelian Inheritance in Man (OMIM)
• PharmGKB