Cns gene therapy: present developments and emerging trends accelerating industry-academia pathways 2

HIGHLIGHTS

• who: Deglon Nicole from the University Hospital (CHUV) Switzerland have published the article: CNS gene therapy: present developments and emerging trends accelerating industry-academia pathways 2, in the Journal: (JOURNAL)
• what: The time between 2000 and 2010 Neurometabolic diseases Lysosomal storage disorders (LSDs) are the major focus of current gene therapy pipeline for inherited neurometabolic diseases, including gangliosidoses, mucopolysaccharidoses (MPS) and metachromatic leukodystrophy.

SUMMARY

For CNS indications, the first gene therapy drugs to, and LibmeldyÒ (ex vivo HSPC gene therapy) for metachromatic 12.The most recent marketing approval (August 2022) was . . .

 



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