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Publications by Alejandro Garanto

ABCA4-associated Disease as a Model for Missing Heritability in Autosomal Recessive Disorders: Novel Noncoding Splice, Cis-Regulatory, Structural, and Recurrent Hypomorphic Variants

Genetics in Medicine

English

Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human C.2991+1655A>G LCA10 Models

Molecular Therapy - Nucleic Acids

Molecular Medicine

English