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Your search keyword '"Alejandro Garanto"' showing total 4 results
Start Over "Alejandro Garanto" Publication Type Magazines Journal genomics & genetics weekly
4 results on '"Alejandro Garanto"'

1. Recent Findings from Radboud University Medical Center (Radboudumc) Highlight Research in Antisense Technology (Efficacy, biodistribution and safety comparison of chemically modified antisense oligonucleotides in the retina).

Subjects
HUMAN genetics, NUCLEIC acids, ACADEMIC medical centers, REPORTERS & reporting, RETINAL diseases
Abstract

A recent study conducted at Radboud University Medical Center in the Netherlands explored the use of antisense oligonucleotides (AONs) for treating inherited retinal diseases. The study compared the efficacy, biodistribution, and safety of AONs with different chemical modifications commonly used in a clinical setting. The results showed that AONs with 2'O-methyl-phosphorothioate (2-OMe/PS) and 2'O-methoxyethyl-phosphoriate (2-MOE/PS) modifications had comparable efficacy and safety profiles, while octa-guanidine-dendrimer-conjugated in vivo PMO-oligonucleotides (ivPMO) caused toxicity. The study provides important insights for future clinical applications and suggests the 2'MOE/PS modification as the most efficacious in these conditions. [Extracted from the article]

Published
2024

2. Radboud University Medical Center Researchers Discuss Findings in Gene Editing (CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids).

Subjects
GENE expression, GENOME editing, ACADEMIC medical centers, NONSENSE mutation, CRISPRS
Abstract

Biotechnology, CRISPR, Eye Diseases and Conditions, Gene Editing, Genetics, Genome Editing, Health and Medicine, Retinal Diseases and Conditions Keywords: Biotechnology; CRISPR; Eye Diseases and Conditions; Gene Editing; Genetics; Genome Editing; Health and Medicine; Retinal Diseases and Conditions EN Biotechnology CRISPR Eye Diseases and Conditions Gene Editing Genetics Genome Editing Health and Medicine Retinal Diseases and Conditions 1033 1033 1 06/19/23 20230623 NES 230623 2023 JUN 23 (NewsRx) -- By a News Reporter-Staff News Editor at Genomics & Genetics Weekly -- Research findings on gene editing are discussed in a new report. [Extracted from the article]

Published
2023

3. Research Data from Radboud University Medical Center Update Understanding of Molecular Science (In Vitro Skeletal Muscle Model of PGM1 Deficiency Reveals Altered Energy Homeostasis).

Subjects
ACADEMIC medical centers, SKELETAL muscle, HOMEOSTASIS
Abstract

No difference was found for steady-state levels of nucleotide sugars, while dynamic flux analysis based on SP 13 sp C6-galactose suggested a block in the use of galactose for energy production in knockout myoblasts." Keywords: Cells; Molecular Science; Myoblasts; Science EN Cells Molecular Science Myoblasts Science 1096 1096 1 05/29/23 20230602 NES 230602 2023 JUN 2 (NewsRx) -- By a News Reporter-Staff News Editor at Genomics & Genetics Weekly -- Data detailed on molecular science have been presented. [Extracted from the article]

Published
2023

4. Researchers from Radboud University Nijmegen Report on Findings in Stargardt Disease [Correction of the Splicing Defect Caused By a Recurrent Variant In Abca4 (C.769-784c<t) That Underlies Stargardt Disease].

Abstract

The article discusses research from Radboud University Nijmegen in the Netherlands which demonstrated the correction of pseudoexon inclusion splicing defect caused by a recurrent gene variant in ABCA4 (C.769-784c>t) that underlies Stargardt disease by using nine antisense oligonucleotides.

Published
2023

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