Format
Sort by
Items per page

Send to

Choose Destination

Search results

Items: 1 to 20 of 82

1.

CRISPR-Generated Animal Models of Duchenne Muscular Dystrophy.

Lim KRQ, Nguyen Q, Dzierlega K, Huang Y, Yokota T.

Genes (Basel). 2020 Mar 24;11(3). pii: E342. doi: 10.3390/genes11030342. Review.

2.

Genome Editing for the Understanding and Treatment of Inherited Cardiomyopathies.

Nguyen Q, Lim KRQ, Yokota T.

Int J Mol Sci. 2020 Jan 22;21(3). pii: E733. doi: 10.3390/ijms21030733. Review.

3.

DUX4 Signalling in the Pathogenesis of Facioscapulohumeral Muscular Dystrophy.

Lim KRQ, Nguyen Q, Yokota T.

Int J Mol Sci. 2020 Jan 22;21(3). pii: E729. doi: 10.3390/ijms21030729. Review.

4.

Amelioration of intracellular Ca2+ regulation by exon-45 skipping in Duchenne muscular dystrophy-induced pluripotent stem cell-derived cardiomyocytes.

Sato M, Shiba N, Miyazaki D, Shiba Y, Echigoya Y, Yokota T, Takizawa H, Aoki Y, Takeda S, Nakamura A.

Biochem Biophys Res Commun. 2019 Nov 26;520(1):179-185. doi: 10.1016/j.bbrc.2019.09.095. Epub 2019 Oct 1.

5.
6.

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

Echigoya Y, Lim KRQ, Melo D, Bao B, Trieu N, Mizobe Y, Maruyama R, Mamchaoui K, Tanihata J, Aoki Y, Takeda S, Mouly V, Duddy W, Yokota T.

Mol Ther. 2019 Nov 6;27(11):2005-2017. doi: 10.1016/j.ymthe.2019.07.012. Epub 2019 Jul 26.

PMID:
31416775
7.

Current understanding and treatment of cardiac and skeletal muscle pathology in laminin-α2 chain-deficient congenital muscular dystrophy.

Nguyen Q, Lim KRQ, Yokota T.

Appl Clin Genet. 2019 Jul 3;12:113-130. doi: 10.2147/TACG.S187481. eCollection 2019.

8.

Antisense oligonucleotides for the treatment of cardiomyopathy in Duchenne muscular dystrophy.

Nguyen Q, Yokota T.

Am J Transl Res. 2019 Mar 15;11(3):1202-1218. eCollection 2019. Review.

9.

Scavenger Receptor Class A1 Mediates Uptake of Morpholino Antisense Oligonucleotide into Dystrophic Skeletal Muscle.

Miyatake S, Mizobe Y, Tsoumpra MK, Lim KRQ, Hara Y, Shabanpoor F, Yokota T, Takeda S, Aoki Y.

Mol Ther Nucleic Acids. 2019 Mar 1;14:520-535. doi: 10.1016/j.omtn.2019.01.008. Epub 2019 Jan 25.

10.

Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic Approaches.

Shimizu-Motohashi Y, Komaki H, Motohashi N, Takeda S, Yokota T, Aoki Y.

J Pers Med. 2019 Jan 7;9(1). pii: E1. doi: 10.3390/jpm9010001. Review.

11.

Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and Challenges.

Echigoya Y, Lim KRQ, Nakamura A, Yokota T.

J Pers Med. 2018 Dec 7;8(4). pii: E41. doi: 10.3390/jpm8040041. Review.

12.

Applications of CRISPR/Cas9 for the Treatment of Duchenne Muscular Dystrophy.

Lim KRQ, Yoon C, Yokota T.

J Pers Med. 2018 Nov 24;8(4). pii: E38. doi: 10.3390/jpm8040038. Review.

13.

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

Lim KRQ, Echigoya Y, Nagata T, Kuraoka M, Kobayashi M, Aoki Y, Partridge T, Maruyama R, Takeda S, Yokota T.

Mol Ther. 2019 Jan 2;27(1):76-86. doi: 10.1016/j.ymthe.2018.10.011. Epub 2018 Oct 19.

14.

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

Lee JJA, Maruyama R, Duddy W, Sakurai H, Yokota T.

Mol Ther Nucleic Acids. 2018 Dec 7;13:596-604. doi: 10.1016/j.omtn.2018.10.004. Epub 2018 Oct 11.

15.

"Of Mice and Measures": A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic.

Gordish-Dressman H, Willmann R, Dalle Pazze L, Kreibich A, van Putten M, Heydemann A, Bogdanik L, Lutz C, Davies K, Demonbreun AR, Duan D, Elsey D, Fukada SI, Girgenrath M, Patrick Gonzalez J, Grounds MD, Nichols A, Partridge T, Passini M, Sanarica F, Schnell FJ, Wells DJ, Yokota T, Young CS, Zhong Z, Spurney C, Spencer M, De Luca A, Nagaraju K, Aartsma-Rus A.

J Neuromuscul Dis. 2018;5(4):407-417. doi: 10.3233/JND-180324.

16.

Exon Skipping Using Antisense Oligonucleotides for Laminin-Alpha2-Deficient Muscular Dystrophy.

Hara Y, Mizobe Y, Miyatake S, Takizawa H, Nagata T, Yokota T, Takeda S, Aoki Y.

Methods Mol Biol. 2018;1828:553-564. doi: 10.1007/978-1-4939-8651-4_36.

PMID:
30171567
17.

Morpholino-Mediated Exon Skipping Targeting Human ACVR1/ALK2 for Fibrodysplasia Ossificans Progressiva.

Maruyama R, Yokota T.

Methods Mol Biol. 2018;1828:497-502. doi: 10.1007/978-1-4939-8651-4_32.

PMID:
30171563
18.

Systemic and ICV Injections of Antisense Oligos into SMA Mice and Evaluation.

Aslesh T, Maruyama R, Yokota T.

Methods Mol Biol. 2018;1828:455-465. doi: 10.1007/978-1-4939-8651-4_28.

PMID:
30171559
19.

In Vitro Evaluation of Antisense-Mediated Exon Inclusion for Spinal Muscular Atrophy.

Touznik A, Maruyama R, Yokota T.

Methods Mol Biol. 2018;1828:439-454. doi: 10.1007/978-1-4939-8651-4_27.

PMID:
30171558
20.

In Vivo Evaluation of Multiple Exon Skipping with Peptide-PMOs in Cardiac and Skeletal Muscles in Dystrophic Dogs.

Maruyama R, Aoki Y, Takeda S, Yokota T.

Methods Mol Biol. 2018;1828:365-379. doi: 10.1007/978-1-4939-8651-4_23.

PMID:
30171554

Supplemental Content

Loading ...
Support Center