R01 HL130040/HL/NHLBI NIH HHS/United States[Grants and Funding] - Search Results

Year	Number of Results
2016	1
2017	2
2018	5
2019	2
2020	2
2021	3
2022	4
2023	3
2024	0

1

In vivo HSC prime editing rescues sickle cell disease in a mouse model.

Li C, Georgakopoulou A, Newby GA, Chen PJ, Everette KA, Paschoudi K, Vlachaki E, Gil S, Anderson AK, Koob T, Huang L, Wang H, Kiem HP, Liu DR, Yannaki E, Lieber A. Li C, et al. Blood. 2023 Apr 27;141(17):2085-2099. doi: 10.1182/blood.2022018252. Blood. 2023. PMID: 36800642 Free PMC article.

2

In Vivo Hematopoietic Stem Cell Transduction.

Richter M, Stone D, Miao C, Humbert O, Kiem HP, Papayannopoulou T, Lieber A. Richter M, et al. Hematol Oncol Clin North Am. 2017 Oct;31(5):771-785. doi: 10.1016/j.hoc.2017.06.001. Hematol Oncol Clin North Am. 2017. PMID: 28895846 Free PMC article. Review.

3

Safe and Effective In Vivo Targeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development.

Cannon P, Asokan A, Czechowicz A, Hammond P, Kohn DB, Lieber A, Malik P, Marks P, Porteus M, Verhoeyen E, Weissman D, Weissman I, Kiem HP. Cannon P, et al. Hum Gene Ther. 2021 Jan;32(1-2):31-42. doi: 10.1089/hum.2020.263. Hum Gene Ther. 2021. PMID: 33427035 Free PMC article.

4

In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathies.

Li C, Georgakopoulou A, Newby GA, Everette KA, Nizamis E, Paschoudi K, Vlachaki E, Gil S, Anderson AK, Koob T, Huang L, Wang H, Kiem HP, Liu DR, Yannaki E, Lieber A. Li C, et al. JCI Insight. 2022 Oct 10;7(19):e162939. doi: 10.1172/jci.insight.162939. JCI Insight. 2022. PMID: 36006707 Free PMC article.

5

Multiplex Base Editing to Protect from CD33-Directed Therapy: Implications for Immune and Gene Therapy.

Borot F, Humbert O, Newby GA, Fields E, Kohli S, Radtke S, Laszlo GS, Mayuranathan T, Ali AM, Weiss MJ, Yen JS, Walter RB, Liu DR, Mukherjee S, Kiem HP. Borot F, et al. bioRxiv [Preprint]. 2023 Feb 23:2023.02.23.529353. doi: 10.1101/2023.02.23.529353. bioRxiv. 2023. PMID: 36865281 Free PMC article. Preprint.

6

In Vivo Hematopoietic Stem Cell Gene Therapy for SARS-CoV2 Infection Using a Decoy Receptor.

Wang H, Li C, Obadan AO, Frizzell H, Hsiang TY, Gil S, Germond A, Fountain C, Baldessari A, Roffler S, Kiem HP, Fuller DH, Lieber A. Wang H, et al. Hum Gene Ther. 2022 Apr;33(7-8):389-403. doi: 10.1089/hum.2021.295. Hum Gene Ther. 2022. PMID: 35057635 Free PMC article.

7

HDAd6/35++ - A new helper-dependent adenovirus vector platform for in vivo transduction of hematopoietic stem cells.

Wang H, Georgakopoulou A, Zhang W, Kim J, Gil S, Ehrhardt A, Lieber A. Wang H, et al. Mol Ther Methods Clin Dev. 2023 Mar 21;29:213-226. doi: 10.1016/j.omtm.2023.03.008. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37081854 Free PMC article.

8

Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors.

Li C, Wang H, Gil S, Germond A, Fountain C, Baldessari A, Kim J, Liu Z, Georgakopoulou A, Radtke S, Raskó T, Pande A, Chiang C, Chin E, Yannaki E, Izsvák Z, Papayannopoulou T, Kiem HP, Lieber A. Li C, et al. Mol Ther Methods Clin Dev. 2021 Dec 6;24:127-141. doi: 10.1016/j.omtm.2021.12.003. eCollection 2022 Mar 10. Mol Ther Methods Clin Dev. 2021. PMID: 35036470 Free PMC article.

9

Single-dose MGTA-145/plerixafor leads to efficient mobilization and in vivo transduction of HSCs with thalassemia correction in mice.

Li C, Goncalves KA, Raskó T, Pande A, Gil S, Liu Z, Izsvák Z, Papayannopoulou T, Davis JC, Kiem HP, Lieber A. Li C, et al. Blood Adv. 2021 Mar 9;5(5):1239-1249. doi: 10.1182/bloodadvances.2020003714. Blood Adv. 2021. PMID: 33646305 Free PMC article.

10

In vivo HSC transduction in rhesus macaques with an HDAd5/3+ vector targeting desmoglein 2 and transiently overexpressing cxcr4.

Wang H, Germond A, Li C, Gil S, Kim J, Kiem HP, Lieber A. Wang H, et al. Blood Adv. 2022 Aug 9;6(15):4360-4372. doi: 10.1182/bloodadvances.2022007975. Blood Adv. 2022. PMID: 35679480 Free PMC article.

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