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doi: 10.3390/ijms20030649 75

Engineering adeno-associated virus vectors for gene therapy

[DOI] [PMC free article] [PubMed] [Google Scholar] 210.Tapfumaneyi P., Imran M., Mohammed Y., Roberts M.S

Genetic analysis has also revealed primary aberrance in several ER homeostasis-associated genes, including anterior gradient protein 2 homolog, X-box binding protein 1 and sphingolipid biosynthesis regulator 3 in patients with UC (64)

Interestingly, ALS and frontotemporal dementia (FTD) are closely related conditions and often share both genetic and disease mechanisms, especially in people with C9ORF72 mutations suggesting they may be part of the same disease spectrum (Parobkova and Matej, 2021)