胎児の細胞を使うことの問題 その５

Dr Theresa Deisher が、ヒト胎児由来DNAが白血病の原因となる可能性の根拠としている遺伝子療法について

 

https://www.newscientist.com/article/mg22029413-200-bubble-kid-success-puts-gene-therapy-back-on-track/

Trials and tribulations of gene therapy

1990 First approved gene therapy trial. Immune cells from 4-year-old Ashanti DeSilva are given working versions of the ADA gene to treat severe combined immunodeficiency disorder (SCID). It works, but only temporarily.

1992 ADA-SCID is successfully treated through gene therapy on stem cells harvested from bone marrow.

1999 18-year-old Jesse Gelsinger dies following an immune reaction to the virus vector used to insert the corrected gene. US Food and Drug Administration suspends several trials pending re-evaluation of ethical and procedural practices.

2000 Announcement that two boys in France with X-linked SCID or “bubble boy” disease have been cured using gene therapy.

2002 French SCID trial suspended after four children develop leukaemia as a result of the retrovirus vector.

レトロウイルスベクター治療の結果、4人の小児が白血病を発症した後に、フランスのSCID治験は中止された

(Dr Deisher によると、9人中4人が4年以内に発症）

2003 Chinese company Shenzhen SiBiono GeneTech gains approval for treating head and neck cancer with Gendicine, a modified adenovirus carrying a tumour-suppressor gene.

2003 Researchers in the US begin the first human trial using a modified lentivirus (pictured below). It is a disabled HIV virus carrying a gene to inhibit replication. Trial is a success.

2009 Eight-year-old Corey Haas, who has a rare inherited eye disease and is almost blind, gains normal vision following gene therapy to replace a retinal pigment protein.

2009 Progression of the degenerative disease adrenoleukodystrophy is halted in two boys using gene therapy.

2010 An adult with blood disorder beta-thalassaemia no longer needs monthly blood transfusions following gene therapy to insert a corrected beta-globin gene into stem cells that make blood.

2011 Six people with clotting disorder haemophilia B see a reduction in symptoms after gene therapy on liver cells.

2012 Glybera becomes the first gene therapy drug to be approved in the West, with European approval to treat lipoprotein lipase deficiency.

2013 Two papers describe the treatment of children with a degenerative disorder called metachromatic leukodystrophy and immune disorder Wiskott-Aldrich syndrome using gene therapy (Science, doi.org/pnv; doi.org/ppk).

This week Announcement that five children with ADA-SCID have been successfully treated and are doing well.