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Rescue of common and rare exon 2 skipping variants of the GAA gene using modified U1 snRNA.

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Title: Rescue of common and rare exon 2 skipping variants of the GAA gene using modified U1 snRNA.
Authors: Peruzzo P; Regional Coordinator Centre for Rare Diseases, University Hospital of Udine, P. Le Santa Maria Della Misericordi 15, 33100, Udine, Italy., Bergamin N; Regional Coordinator Centre for Rare Diseases, University Hospital of Udine, P. Le Santa Maria Della Misericordi 15, 33100, Udine, Italy., Bon M; Regional Coordinator Centre for Rare Diseases, University Hospital of Udine, P. Le Santa Maria Della Misericordi 15, 33100, Udine, Italy., Cappelli S; International Centre for Genetic Engineering and Biotechnology (ICGEB), Area Science Park, Trieste, Italy., Longo A; International Centre for Genetic Engineering and Biotechnology (ICGEB), Area Science Park, Trieste, Italy., Goina E; International Centre for Genetic Engineering and Biotechnology (ICGEB), Area Science Park, Trieste, Italy., Stuani C; International Centre for Genetic Engineering and Biotechnology (ICGEB), Area Science Park, Trieste, Italy., Buratti E; International Centre for Genetic Engineering and Biotechnology (ICGEB), Area Science Park, Trieste, Italy., Dardis A; Regional Coordinator Centre for Rare Diseases, University Hospital of Udine, P. Le Santa Maria Della Misericordi 15, 33100, Udine, Italy. andrea.dardis@asufc.sanita.fvg.it.
Source: Molecular medicine (Cambridge, Mass.) [Mol Med] 2025 Feb 04; Vol. 31 (1), pp. 45. Date of Electronic Publication: 2025 Feb 04.
Publication Type: Journal Article
Journal Info: Publisher: BioMed Central Country of Publication: England NLM ID: 9501023 Publication Model: Electronic Cited Medium: Internet ISSN: 1528-3658 (Electronic) Linking ISSN: 10761551 NLM ISO Abbreviation: Mol Med Subsets: MEDLINE
Database: MEDLINE Ultimate
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Description
ISSN:1528-3658
DOI:10.1186/s10020-025-01090-z