8 results on '"Alejandro Garanto"'
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2. Generation of hiPSC lines from four glutaric aciduria type I (GA1) patients carrying pathogenic biallelic variants in GCDH
3. Preclinical Development of Antisense Oligonucleotides to Rescue Aberrant Splicing Caused by an Ultrarare ABCA4 Variant in a Child with Early-Onset Stargardt Disease
4. Efficacy, biodistribution and safety comparison of chemically modified antisense oligonucleotides in the retina.
5. Antisense Oligonucleotide-Based Rescue of Complex Intronic Splicing Defects in ABCA4.
6. Preclinical Development of Antisense Oligonucleotides to Rescue Aberrant Splicing Caused by an Ultrarare ABCA4 Variant in a Child with Early-Onset Stargardt Disease.
7. Understanding and Rescuing the Splicing Defect Caused by the Frequent ABCA4 Variant c.4253+43G>A Underlying Stargardt Disease.
8. Abstracts from the 56th European Society of Human Genetics (ESHG) Conference: Oral Presentations
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