Rika Maruyama

University of Alberta

H指数:24

被引数:2,082

个人简介

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Single-base 2'OMe-modified LNA and MOE gapmers selectively silence ACVR1R206H in FOP, improving muscle and tendon bioavailability and safety

单碱基2'OMe修饰的LNA和MOE gapmer选择性沉默FOP中ACVR1R206H，改善肌肉和肌腱的生物利用度与安全性

Molecular Therapy Nucleic Acids

IF6.1

2026-04-160

OAAI

DG9 boosts PMO nuclear uptake and exon skipping to restore dystrophic muscle and cardiac function

DG9 通过提高 PMO 核摄取和外显子跳读来恢复肌营养不良骨骼肌和心脏功能

NATURE COMMUNICATIONS

IF15.7

2025-05-140

AOC 1044 induces exon 44 skipping and restores dystrophin protein in preclinical models of Duchenne muscular dystrophy

AOC 1044诱导外显子44的跳跃，并在杜氏肌营养不良症的临床前模型中恢复了抗肌萎缩蛋白的表达。

NUCLEIC ACIDS RESEARCH

IF13.1

2025-04-040

PRE

Antisense oligonucleotide-mediated exon 27 skipping restores dysferlin function in dysferlinopathy patient-derived muscle cells

MOLECULAR THERAPY NUCLEIC ACIDS

IF6.1

2025-03-010

OAAI

DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration

JCI INSIGHT

IF6.1

2023-03-088

Allele-Selective Locked Nucleic Acid Gapmers for the Treatment of Fibrodysplasia Ossificans Progressiva Knock Down the Pathogenic ACVR1R206H Transcript and Inhibit Osteogenic Differentiation

NUCLEIC ACID THERAPEUTICS

IF4.7

2022-06-018

PRE

Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

IF9.1

2022-02-2223

OAAI

eSkip-Finder: a machine learning-based web application and database to identify the optimal sequences of antisense oligonucleotides for exon skipping

NUCLEIC ACIDS RESEARCH

IF13.1

2021-06-096

OAAI

Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides

NUCLEIC ACID THERAPEUTICS

IF4.7

2021-04-019

OAAI

DUX4 Transcript Knockdown with Antisense 2′-O-Methoxyethyl Gapmers for the Treatment of Facioscapulohumeral Muscular Dystrophy

MOLECULAR THERAPY

IF12

2021-02-0124

OAAI

A Genotype-Phenotype Correlation Study of Exon Skip-Equivalent In-Frame Deletions and Exon Skip-Amenable Out-of-Frame Deletions across the DMD Gene to Simulate the Effects of Exon-Skipping Therapies: A Meta-Analysis

JOURNAL OF PERSONALIZED MEDICINE

IF3

2021-01-1417

OAAI

Molecular Diagnosis and Novel Therapies for Neuromuscular Diseases

JOURNAL OF PERSONALIZED MEDICINE

IF3

2020-09-163

OAAI

Inhibition of DUX4 expression with antisense LNA gapmers as a therapy for facioscapulohumeral muscular dystrophy

反义LNA gapmers抑制DUX4表达治疗面肩肱型肌营养不良症

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

IF9.1

2020-06-2940

Exons 45-55 Skipping Using Mutation-Tailored Cocktails of Antisense Morpholinos in the DMD Gene

MOLECULAR THERAPY

IF12

2019-11-0140

OAAI

Efficacy of Multi-exon Skipping Treatment in Duchenne Muscular Dystrophy Dog Model Neonates

MOLECULAR THERAPY

IF12

2019-01-0123

OAAI

Identification of Novel Antisense-Mediated Exon Skipping Targets in DYSF for Therapeutic Treatment of Dysferlinopathy

MOLECULAR THERAPY-NUCLEIC ACIDS

IF0

2018-12-0130

OAAI

Skipping Multiple Exons to Treat DMD-Promises and Challenges

BIOMEDICINES

IF3.9

2018-01-0240

OAAI

Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy

MOLECULAR THERAPY

IF12

2017-11-0172

OAAI

LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts

SCIENTIFIC REPORTS

IF3.9

2017-06-1641

OAAI

Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

IF9.1

2017-04-0391

OAAI

Rika Maruyama

个人简介

教育背景

工作经历

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