Burghes AHM - Search Results

1

Translation reinitiation after uORFs does not fully protect mRNAs from nonsense-mediated decay.

Russell PJ, Slivka JA, Boyle EP, Burghes AHM, Kearse MG. Russell PJ, et al. Among authors: burghes ahm. RNA. 2023 Jun;29(6):735-744. doi: 10.1261/rna.079525.122. Epub 2023 Mar 6. RNA. 2023. PMID: 36878710 Free PMC article.

2

In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans.

Schwartz MK, Likhite S, Vetter TA, Baird MC, McGovern V, Sierra Delgado A, Mendel T, Burghes A, Meyer KC. Schwartz MK, et al. Mol Ther Methods Clin Dev. 2023 May 16;30:16-29. doi: 10.1016/j.omtm.2023.05.016. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37746244 Free PMC article.

3

Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy.

Day JW, Mendell JR, Burghes AHM, van Olden RW, Adhikary RR, Dilly KW. Day JW, et al. Among authors: burghes ahm. Mol Ther Methods Clin Dev. 2023 Sep 20;31:101117. doi: 10.1016/j.omtm.2023.101117. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37822718 Free PMC article.

4

Evaluation of the orally bioavailable 4-phenylbutyrate-tethered trichostatin A analogue AR42 in models of spinal muscular atrophy.

Lumpkin CJ, Harris AW, Connell AJ, Kirk RW, Whiting JA, Saieva L, Pellizzoni L, Burghes AHM, Butchbach MER. Lumpkin CJ, et al. Among authors: burghes ahm. Sci Rep. 2023 Jun 26;13(1):10374. doi: 10.1038/s41598-023-37496-0. Sci Rep. 2023. PMID: 37365234 Free PMC article.

5

Base editing rescue of spinal muscular atrophy in cells and in mice.

Arbab M, Matuszek Z, Kray KM, Du A, Newby GA, Blatnik AJ, Raguram A, Richter MF, Zhao KT, Levy JM, Shen MW, Arnold WD, Wang D, Xie J, Gao G, Burghes AHM, Liu DR. Arbab M, et al. Among authors: burghes ahm. Science. 2023 Apr 21;380(6642):eadg6518. doi: 10.1126/science.adg6518. Epub 2023 Apr 14. Science. 2023. PMID: 36996170 Free PMC article.

6

Retraction Note: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN.

Foust KD, Wang X, McGovern VL, Braun L, Bevan AK, Haidet AM, Le TT, Morales PR, Rich MM, Burghes AHM, Kaspar BK. Foust KD, et al. Among authors: burghes ahm. Nat Biotechnol. 2022 Nov;40(11):1692. doi: 10.1038/s41587-022-01497-7. Nat Biotechnol. 2022. PMID: 36203015 Free PMC article. No abstract available.

7

Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue.

Thomsen G, Burghes AHM, Hsieh C, Do J, Chu BTT, Perry S, Barkho B, Kaufmann P, Sproule DM, Feltner DE, Chung WK, McGovern VL, Hevner RF, Conces M, Pierson CR, Scoto M, Muntoni F, Mendell JR, Foust KD. Thomsen G, et al. Among authors: burghes ahm. Nat Med. 2021 Oct;27(10):1701-1711. doi: 10.1038/s41591-021-01483-7. Epub 2021 Oct 4. Nat Med. 2021. PMID: 34608334 Clinical Trial.

8

Persistent neuromuscular junction transmission defects in adults with spinal muscular atrophy treated with nusinersen.

Arnold WD, Severyn S, Zhao S, Kline D, Linsenmayer M, Kelly K, Tellez M, Bartlett A, Heintzman S, Reynolds J, Sterling G, Weaver T, Rajneesh K, Burghes AHM, Kolb SJ, Elsheikh B. Arnold WD, et al. Among authors: burghes ahm. BMJ Neurol Open. 2021 Aug 12;3(2):e000164. doi: 10.1136/bmjno-2021-000164. eCollection 2021. BMJ Neurol Open. 2021. PMID: 34466806 Free PMC article.

9

What Genetics Has Told Us and How It Can Inform Future Experiments for Spinal Muscular Atrophy, a Perspective.

Blatnik AJ 3rd, McGovern VL, Burghes AHM. Blatnik AJ 3rd, et al. Among authors: burghes ahm. Int J Mol Sci. 2021 Aug 6;22(16):8494. doi: 10.3390/ijms22168494. Int J Mol Sci. 2021. PMID: 34445199 Free PMC article.

10

Dual SMN inducing therapies can rescue survival and motor unit function in symptomatic ∆7SMA mice.

Kray KM, McGovern VL, Chugh D, Arnold WD, Burghes AHM. Kray KM, et al. Among authors: burghes ahm. Neurobiol Dis. 2021 Nov;159:105488. doi: 10.1016/j.nbd.2021.105488. Epub 2021 Aug 20. Neurobiol Dis. 2021. PMID: 34425216 Free PMC article.

Save citations to file

Email citations

Send citations to clipboard

Add to Collections

Add to My Bibliography

Create a file for external citation management software

Your saved search

Your RSS Feed

My NCBI Filters

Text availability

Article attribute

Article type

Publication date

72 results

Search Page