Whittaker MN - Search Results

1

High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease.

Bzhilyanskaya V, Ma L, Liu S, Fox LR, Whittaker MN, Meis RJ, Choi U, Lawson A, Ma M, Theobald N, Burkett S, Sweeney CL, Lazzarotto CR, Tsai SQ, Lack JB, Wu X, Dahl GA, Malech HL, Kleinstiver BP, De Ravin SS. Bzhilyanskaya V, et al. Among authors: whittaker mn. Sci Transl Med. 2024 Oct 16;16(769):eadj6779. doi: 10.1126/scitranslmed.adj6779. Epub 2024 Oct 16. Sci Transl Med. 2024. PMID: 39413163

2

Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants.

Walton RT, Christie KA, Whittaker MN, Kleinstiver BP. Walton RT, et al. Among authors: whittaker mn. Science. 2020 Apr 17;368(6488):290-296. doi: 10.1126/science.aba8853. Epub 2020 Mar 26. Science. 2020. PMID: 32217751 Free PMC article.

3

Allele-specific silencing of the gain-of-function mutation in Huntington's disease using CRISPR/Cas9.

Shin JW, Hong EP, Park SS, Choi DE, Seong IS, Whittaker MN, Kleinstiver BP, Chen RZ, Lee JM. Shin JW, et al. Among authors: whittaker mn. JCI Insight. 2022 Oct 10;7(19):e141042. doi: 10.1172/jci.insight.141042. JCI Insight. 2022. PMID: 36040815 Free PMC article.

4

Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo.

Tao Y, Lamas V, Du W, Zhu W, Li Y, Whittaker MN, Zuris JA, Thompson DB, Rameshbabu AP, Shu Y, Gao X, Hu JH, Pei C, Kong WJ, Liu X, Wu H, Kleinstiver BP, Liu DR, Chen ZY. Tao Y, et al. Among authors: whittaker mn. Nat Commun. 2023 Aug 15;14(1):4928. doi: 10.1038/s41467-023-40476-7. Nat Commun. 2023. PMID: 37582836 Free PMC article.

5

A base editing strategy using mRNA-LNPs for in vivo correction of the most frequent phenylketonuria variant.

Brooks DL, Whittaker MN, Said H, Dwivedi G, Qu P, Musunuru K, Ahrens-Nicklas RC, Alameh MG, Wang X. Brooks DL, et al. Among authors: whittaker mn. HGG Adv. 2024 Jan 11;5(1):100253. doi: 10.1016/j.xhgg.2023.100253. Epub 2023 Nov 2. HGG Adv. 2024. PMID: 37922902 Free PMC article.

6

Efficient in vivo prime editing corrects the most frequent phenylketonuria variant, associated with high unmet medical need.

Brooks DL, Whittaker MN, Qu P, Musunuru K, Ahrens-Nicklas RC, Wang X. Brooks DL, et al. Among authors: whittaker mn. Am J Hum Genet. 2023 Dec 7;110(12):2003-2014. doi: 10.1016/j.ajhg.2023.10.005. Epub 2023 Nov 3. Am J Hum Genet. 2023. PMID: 37924808 Free PMC article.

7

Epigenome Editing Durability Varies Widely Across Cardiovascular Disease Target Genes.

Whittaker MN, Testa LC, Quigley A, Jindal I, Cortez-Alvarado SV, Qu P, Yang Y, Alameh MG, Musunuru K, Wang X. Whittaker MN, et al. Arterioscler Thromb Vasc Biol. 2023 Oct;43(10):2075-2077. doi: 10.1161/ATVBAHA.123.319748. Epub 2023 Aug 17. Arterioscler Thromb Vasc Biol. 2023. PMID: 37589141 Free PMC article. No abstract available.

8

Therapeutic application of genome editing in dyslipidemia.

Whittaker MN, Musunuru K. Whittaker MN, et al. Curr Opin Lipidol. 2022 Apr 1;33(2):133-138. doi: 10.1097/MOL.0000000000000805. Curr Opin Lipidol. 2022. PMID: 34907967 Review.

9

Epigenome Editing Durability Varies Widely Across Cardiovascular Disease Target Genes.

Whittaker MN, Testa LC, Quigley A, Jindal I, Cortez-Alvarado SV, Qu P, Yang Y, Alameh MG, Musunuru K, Wang X. Whittaker MN, et al. bioRxiv [Preprint]. 2023 May 17:2023.05.17.541156. doi: 10.1101/2023.05.17.541156. bioRxiv. 2023. Update in: Arterioscler Thromb Vasc Biol. 2023 Oct;43(10):2075-2077. doi: 10.1161/ATVBAHA.123.319748 PMID: 37292627 Free PMC article. Updated. Preprint.

10

Improved specificity and efficacy of base-editing therapies with hybrid guide RNAs.

Whittaker MN, Brooks DL, Quigley A, Jindal I, Said H, Qu P, Wang JZ, Ahrens-Nicklas RC, Musunuru K, Alameh MG, Peranteau WH, Wang X. Whittaker MN, et al. bioRxiv [Preprint]. 2024 Jun 16:2024.04.22.590531. doi: 10.1101/2024.04.22.590531. bioRxiv. 2024. PMID: 38712058 Free PMC article. Preprint.

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