R01 DK098252/DK/NIDDK NIH HHS/United States[Grants and Funding] - Search Results

Year	Number of Results
2013	1
2014	3
2015	2
2017	4
2018	3
2019	8
2020	1
2023	1
2024	1

1

Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

Keeler AM, Flotte TR. Keeler AM, et al. Annu Rev Virol. 2019 Sep 29;6(1):601-621. doi: 10.1146/annurev-virology-092818-015530. Epub 2019 Jul 5. Annu Rev Virol. 2019. PMID: 31283441 Free PMC article.

2

Lack of acute xenogeneic graft- versus-host disease, but retention of T-cell function following engraftment of human peripheral blood mononuclear cells in NSG mice deficient in MHC class I and II expression.

Brehm MA, Kenney LL, Wiles MV, Low BE, Tisch RM, Burzenski L, Mueller C, Greiner DL, Shultz LD. Brehm MA, et al. FASEB J. 2019 Mar;33(3):3137-3151. doi: 10.1096/fj.201800636R. Epub 2018 Nov 1. FASEB J. 2019. PMID: 30383447 Free PMC article.

3

Gene Therapy 2017: Progress and Future Directions.

Keeler AM, ElMallah MK, Flotte TR. Keeler AM, et al. Clin Transl Sci. 2017 Jul;10(4):242-248. doi: 10.1111/cts.12466. Epub 2017 May 23. Clin Transl Sci. 2017. PMID: 28383804 Free PMC article. Review. No abstract available.

4

Prime Editing: A Novel Cas9-Reverse Transcriptase Fusion May Revolutionize Genome Editing.

Flotte TR, Gao G. Flotte TR, et al. Hum Gene Ther. 2019 Dec;30(12):1445-1446. doi: 10.1089/hum.2019.29098.trf. Hum Gene Ther. 2019. PMID: 31860398 No abstract available.

5

Type 1 diabetes contributes to combined pulmonary fibrosis and emphysema in male alpha 1 antitrypsin deficient mice.

Park SS, Mai M, Ploszaj M, Cai H, McGarvey L, Mueller C, Garcia-Arcos I, Geraghty P. Park SS, et al. PLoS One. 2023 Oct 11;18(10):e0291948. doi: 10.1371/journal.pone.0291948. eCollection 2023. PLoS One. 2023. PMID: 37819895 Free PMC article.

6

Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques.

Blackwood M, Gruntman AM, Tang Q, Pires-Ferreira D, Reil D, Kondratov O, Marsic D, Zolotukhin S, Gernoux G, Keeler AM, Mueller C, Flotte TR. Blackwood M, et al. Mol Ther Methods Clin Dev. 2024 Jan 30;32(1):101200. doi: 10.1016/j.omtm.2024.101200. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38445045 Free PMC article.

7

Recombinant AAV as a platform for translating the therapeutic potential of RNA interference.

Borel F, Kay MA, Mueller C. Borel F, et al. Mol Ther. 2014 Apr;22(4):692-701. doi: 10.1038/mt.2013.285. Epub 2013 Dec 19. Mol Ther. 2014. PMID: 24352214 Free PMC article. Review.

8

AAV9 gene replacement therapy for respiratory insufficiency in very-long chain acyl-CoA dehydrogenase deficiency.

Zieger M, Keeler AM, Flotte TR, ElMallah MK. Zieger M, et al. J Inherit Metab Dis. 2019 Sep;42(5):870-877. doi: 10.1002/jimd.12101. Epub 2019 May 3. J Inherit Metab Dis. 2019. PMID: 30993714 Free PMC article.

9

Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans.

Gernoux G, Gruntman AM, Blackwood M, Zieger M, Flotte TR, Mueller C. Gernoux G, et al. Mol Ther. 2020 Mar 4;28(3):747-757. doi: 10.1016/j.ymthe.2020.01.004. Epub 2020 Jan 13. Mol Ther. 2020. PMID: 31982038 Free PMC article.

10

In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency.

Song CQ, Wang D, Jiang T, O'Connor K, Tang Q, Cai L, Li X, Weng Z, Yin H, Gao G, Mueller C, Flotte TR, Xue W. Song CQ, et al. Hum Gene Ther. 2018 Aug;29(8):853-860. doi: 10.1089/hum.2017.225. Epub 2018 May 14. Hum Gene Ther. 2018. PMID: 29597895 Free PMC article.

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