Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy.
Ashish O GuptaGerald RaymondElizabeth I PierpontStephan KempR Scott McIvorArpana RayannavarBradley MillerTroy C LundPaul J OrchardPublished in: Expert opinion on biological therapy (2022)
The data regarding this lentiviral-based gene therapy approach and its relative risks and benefits is still being evaluated. This information is explored in the context of the experience with allogeneic HSCT for cerebral ALD.
Keyphrases
- gene therapy
- stem cell transplantation
- hematopoietic stem cell
- subarachnoid hemorrhage
- bone marrow
- high dose
- cerebral ischemia
- electronic health record
- big data
- brain injury
- stem cells
- healthcare
- cerebral blood flow
- cell therapy
- health information
- blood brain barrier
- risk assessment
- artificial intelligence
- data analysis