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Gene therapy for inherited metabolic diseases

Berna Seker Yilmaz
Berna Seker Yilmaz
, 
Sonam Gurung
Sonam Gurung
, 
Dany Perocheau
Dany Perocheau
, 
John Counsell
John Counsell
 oraz 
Julien Baruteau
Julien Baruteau
   | 10 lis 2020
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Article Category: Review paper
Data publikacji: 10 lis 2020
Zakres stron: 53 - 64
DOI: https://doi.org/10.34763/jmotherandchild.20202402si.2004.000009
Słowa kluczowe
© 2020 Seker Yilmaz et al, published by Sciendo
This work is licensed under the Creative Commons Attribution 4.0 International License.

Figure 1

Transduction pathways of lentiviral and adeno-associated viral (AAV) vectors : cellular uptake and in-cell processing.
Transduction pathways of lentiviral and adeno-associated viral (AAV) vectors : cellular uptake and in-cell processing.

Main features of gene therapy strategies.

Non viral Lentivirus Adenovirus Adeno-associated virus
Derived from pathogenic virus No Yes Yes Yes
Size of transgene No limit 14kb 7.5kb 4.7kb
Insertion to host genome (Integration) No Yes No Rarely
Long-lasting gene expression No Yes Yes Yes
Safety issues No Insertional mutagenesis Immune response Limited immune response

Indicative list of gene therapy clinical trials for inherited metabolic diseases in 2020.

Inherited metabolic diseases subgroup Disease Sponsor Phase Status Vector NCT number (Clinicaltrials.gov)
Glycogen storage disease 1A Ultragenyx I/II R AAV8 NCT035117085
Crigler-Najjar Genethon-Selecta Bio I/II R AAV NCT03466463
Ornithine transcarbamylase deficiency University of Pennsylvania I T Adeno- viral NCT00004498
Ultragenyx I/II R AAV8 NCT02991144
Intermediary metabolism Methylmalonic acidaemia Moderna Therapeutics I/II R Non viral NCT03810690
Propionic acidaemia Moderna Therapeutics I/II A-NR Non viral NCT04159103
Phenylketonuria Homology Medicines I/II R AAVH- SC15 NCT03952156
National Taiwan University Hospital I/II T AAV2 NCT01395641
Aromatic L-amino acid decarboxylase deficiency National Taiwan University Hospital II R AAV2 NCT02926066
National Institute of Health I R AAV2 NCT02852213
Lipid metabolism Homozygous Familial Hypercholesterolaemia RegenX Bio I/II R AAV NCT02651675
Sangamo Therapeutics I/II H AAV6 NCT02702115
Mucopolysaccharidosis 1 RegenX Bio I R AAV9 NCT03580083
Orchard Therapeutics/San Raffaele-Telethon Institute for Gene Therapy I/II R LV NCT03488394
Mucopolysaccharidosis 2 Sangamo Therapeutics I/II H AAV6 NCT03041324
RegenX Bio I/II R AAV9 NCT03566043
Manchester University I/II R LV NCT04201405
Lysogene I/II T AAVrh10 NCT01474343
Mucopolysaccharidosis 3A Lysogene I/II R AAVrh10 NCT03612869
Abeona Therapeutics I/II R AAV9 NCT02716246; NCT04088734
Mucopolysaccharidosis 3B Abeona Therapeutics I/II R AAV9 NTC03315182
Uniqure I/II T AAV5 NCT03300453
Mucopolysaccharidosis 6 Fondazione Telethon I/II R AAV8 NCT03173521
Audentes Therapeutics I/II A-NR AAV8 NCT04174105
Spark Therapeutics I/II A-NR AAV NCT04093349
Lysosomal storage diseases Pompe disease Florida University I/II T AAV1 NCT00976352
Florida University I R AAV9 NCT02240407
Danon disease Rocket Pharmaceuticals I R AAV9 NCT03882437
Sangamo Therapeutics I/II R AAV6 NCT04046224
Fabry disease Freeline Therapeutics I/II R AAV NCT04040049
AvroBio I/II R LV NCT03454893
Ceroide lipofuscinosis 6 Amicus Therapeutics I/II A-NR AAV9 NCT02725580
Ceroide lipofuscinosis 3 Amicus Therapeutics I/II R AAV9 NCT03770572
Cornell University I A-NR AAV.rh10 NCT01161576
Ceroide lipofuscinosis 2 Cornell University I A-NR AAV2 NCT00151216
Cornell University I/II A-NR AAV.rh10 NCT01414985
GM1 Gangliosidosis National Human Genome Research Institute I/II R AAV9 NCT03952637
Metachromatic leukodystrophy Orchard Therapeutics/San Raffaele-Telethon Institute for Gene Therapy I/II R LV NTC03392987
Shenzhen University I/II R LV NCT02559830
Gaucher type 1 AvroBio I/II R LV NCT04145037
Bluebird Bio II/III A-NR LV NCT01896102
Peroxisomal disorders X-linked childhood cerebral adrenoleukodystrophy Bluebird Bio III A-NR LV NCT03852498
Shenzhen Second People's Hospital I/II R LV NCT0372755
eISSN:
2719-535X
Język:
Angielski
Częstotliwość wydawania:
Volume Open
Dziedziny czasopisma:
Medicine, Clinical Medicine, Pediatrics and Juvenile Medicine, Paediatric Haematology and Oncology, Public Health
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