36222125 30120348 37273786 31801211 32637445 32929188 31426500 34130750 30909783 35676652 32829028 30086404 32525760 31098852 30258237 30531949 35805149 34392299 30213863 35977479 31011884 32413282 29333255 32709756 29892259 31357652 34372914 2975677 - Search Results

Year	Number of Results
2017	2
2018	15
2019	19
2020	17
2021	17
2022	10
2023	7

1

Dynamics and variability of transcriptomic dysregulation in congenital myotonic dystrophy during pediatric development.

Hale MA, Bates K, Provenzano M, Johnson NE. Hale MA, et al. Hum Mol Genet. 2023 Apr 20;32(9):1413-1428. doi: 10.1093/hmg/ddac254. Hum Mol Genet. 2023. PMID: 36222125 Free PMC article.

2

C9orf72-mediated ALS and FTD: multiple pathways to disease.

Balendra R, Isaacs AM. Balendra R, et al. Nat Rev Neurol. 2018 Sep;14(9):544-558. doi: 10.1038/s41582-018-0047-2. Nat Rev Neurol. 2018. PMID: 30120348 Free PMC article. Review.

3

Specific DMPK-promoter targeting by CRISPRi reverses myotonic dystrophy type 1-associated defects in patient muscle cells.

Porquet F, Weidong L, Jehasse K, Gazon H, Kondili M, Blacher S, Massotte L, Di Valentin E, Furling D, Gillet NA, Klein AF, Seutin V, Willems L. Porquet F, et al. Mol Ther Nucleic Acids. 2023 May 13;32:857-871. doi: 10.1016/j.omtn.2023.05.007. eCollection 2023 Jun 13. Mol Ther Nucleic Acids. 2023. PMID: 37273786 Free PMC article.

4

Dead Cas Systems: Types, Principles, and Applications.

Brezgin S, Kostyusheva A, Kostyushev D, Chulanov V. Brezgin S, et al. Int J Mol Sci. 2019 Nov 30;20(23):6041. doi: 10.3390/ijms20236041. Int J Mol Sci. 2019. PMID: 31801211 Free PMC article. Review.

5

Unexpected Mutations by CRISPR-Cas9 CTG Repeat Excision in Myotonic Dystrophy and Use of CRISPR Interference as an Alternative Approach.

Ikeda M, Taniguchi-Ikeda M, Kato T, Shinkai Y, Tanaka S, Hagiwara H, Sasaki N, Masaki T, Matsumura K, Sonoo M, Kurahashi H, Saito F. Ikeda M, et al. Mol Ther Methods Clin Dev. 2020 May 22;18:131-144. doi: 10.1016/j.omtm.2020.05.024. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32637445 Free PMC article.

6

The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy type 1.

Batra R, Nelles DA, Roth DM, Krach F, Nutter CA, Tadokoro T, Thomas JD, Sznajder ŁJ, Blue SM, Gutierrez HL, Liu P, Aigner S, Platoshyn O, Miyanohara A, Marsala M, Swanson MS, Yeo GW. Batra R, et al. Nat Biomed Eng. 2021 Feb;5(2):157-168. doi: 10.1038/s41551-020-00607-7. Epub 2020 Sep 14. Nat Biomed Eng. 2021. PMID: 32929188 Free PMC article.

7

Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules.

Reddy K, Jenquin JR, Cleary JD, Berglund JA. Reddy K, et al. Int J Mol Sci. 2019 Aug 17;20(16):4017. doi: 10.3390/ijms20164017. Int J Mol Sci. 2019. PMID: 31426500 Free PMC article. Review.

8

Genetic mutations and molecular mechanisms of Fuchs endothelial corneal dystrophy.

Liu X, Zheng T, Zhao C, Zhang Y, Liu H, Wang L, Liu P. Liu X, et al. Eye Vis (Lond). 2021 Jun 15;8(1):24. doi: 10.1186/s40662-021-00246-2. Eye Vis (Lond). 2021. PMID: 34130750 Free PMC article. Review.

9

Repeat-Associated Non-ATG Translation: Molecular Mechanisms and Contribution to Neurological Disease.

Nguyen L, Cleary JD, Ranum LPW. Nguyen L, et al. Annu Rev Neurosci. 2019 Jul 8;42:227-247. doi: 10.1146/annurev-neuro-070918-050405. Epub 2019 Mar 25. Annu Rev Neurosci. 2019. PMID: 30909783 Free PMC article. Review.

10

A 20-year bibliometric analysis of Fuchs endothelial corneal dystrophy: from 2001 to 2020.

Lin F, Zhang L, Wang Y, Fu D, Wang Y, Zhou X. Lin F, et al. BMC Ophthalmol. 2022 Jun 8;22(1):255. doi: 10.1186/s12886-022-02468-x. BMC Ophthalmol. 2022. PMID: 35676652 Free PMC article.

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