Gene therapy found effective to cure ALD, brain disease which causes brain degeneration among young male patients

Briefing

Gene therapy found effective to cure ALD, brain disease which causes brain degeneration among young male patients

October 9, 2017

Briefing
  • Successful Gene Therapy – Doctors successfully administered gene therapy among test subjects to cure brain disease called adrenoleukodystrophy, or ALD
  • ALD – Caused by mutated gene and results in neurodegeneration, resulting in patients losing ability to walk or talk after few years (disease affects one in every 18,000 people)
  • Test Results – Show 15 out of 17 boys, aged four to 13 years old, that participated in study had no more symptoms after two years
  • How It Works – Bone marrow stem cells extracted from patient before corrected gene is inserted to cells and infused to bone marrow, allowing good genes to multiply and become glial cells that surround and insulate neurons, stopping brain degeneration
  • Disclaimer – Therapy most effective when patient gets treatment before symptoms occur

Accelerator
Biotechnology and Bioinformatics

Sector
Healthcare/Health Sciences

Organization
Boston Children's Hospital, Fundacion Investigar, Harvard Medical School, Hôpital Bicêtre-Hôpitaux Universitaires Paris Sud, Massachusetts General Hospital, University College - London, University College London Great Ormond Street Institute of Child Health, University of California - Los Angeles, University of Minnesota Children’s Hospital, Women’s and Children’s Hospital

Source

Original Publication Date
October 5, 2017

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