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Items: 1 to 20 of 81

1.

In Situ Modification of Tissue Stem and Progenitor Cell Genomes.

Goldstein JM, Tabebordbar M, Zhu K, Wang LD, Messemer KA, Peacker B, Ashrafi Kakhki S, Gonzalez-Celeiro M, Shwartz Y, Cheng JKW, Xiao R, Barungi T, Albright C, Hsu YC, Vandenberghe LH, Wagers AJ.

Cell Rep. 2019 Apr 23;27(4):1254-1264.e7. doi: 10.1016/j.celrep.2019.03.105.

2.

AAV Engineering Identifies a Species Barrier That Highlights a Portal to the Brain.

Vandenberghe LH.

Mol Ther. 2019 May 8;27(5):901-903. doi: 10.1016/j.ymthe.2019.04.006. Epub 2019 Apr 16. No abstract available.

PMID:
31000409
3.

Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality.

Hudry E, Vandenberghe LH.

Neuron. 2019 Apr 3;102(1):263. doi: 10.1016/j.neuron.2019.03.020. No abstract available.

PMID:
30946822
4.

Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality.

Hudry E, Vandenberghe LH.

Neuron. 2019 Mar 6;101(5):839-862. doi: 10.1016/j.neuron.2019.02.017. Review. Erratum in: Neuron. 2019 Apr 3;102(1):263.

PMID:
30844402
5.

Quantitative and Digital Droplet-Based AAV Genome Titration.

Sanmiguel J, Gao G, Vandenberghe LH.

Methods Mol Biol. 2019;1950:51-83. doi: 10.1007/978-1-4939-9139-6_4.

PMID:
30783968
6.

Residues on Adeno-associated Virus Capsid Lumen Dictate Interactions and Compatibility with the Assembly-Activating Protein.

Maurer AC, Cepeda Diaz AK, Vandenberghe LH.

J Virol. 2019 Mar 21;93(7). pii: e02013-18. doi: 10.1128/JVI.02013-18. Print 2019 Apr 1.

PMID:
30651367
7.

FGF21 underlies a hormetic response to metabolic stress in methylmalonic acidemia.

Manoli I, Sysol JR, Epping MW, Li L, Wang C, Sloan JL, Pass A, Gagné J, Ktena YP, Li L, Trivedi NS, Ouattara B, Zerfas PM, Hoffmann V, Abu-Asab M, Tsokos MG, Kleiner DE, Garone C, Cusmano-Ozog K, Enns GM, Vernon HJ, Andersson HC, Grunewald S, Elkahloun AG, Girard CL, Schnermann J, DiMauro S, Andres-Mateos E, Vandenberghe LH, Chandler RJ, Venditti CP.

JCI Insight. 2018 Dec 6;3(23). pii: 124351. doi: 10.1172/jci.insight.124351.

8.

Novel engineered, membrane-localized variants of vascular endothelial growth factor (VEGF) protect retinal ganglion cells: a proof-of-concept study.

Shen J, Xiao R, Bair J, Wang F, Vandenberghe LH, Dartt D, Baranov P, Ng YSE.

Cell Death Dis. 2018 Oct 3;9(10):1018. doi: 10.1038/s41419-018-1049-0.

9.

Efficient Gene Transfer to the Central Nervous System by Single-Stranded Anc80L65.

Hudry E, Andres-Mateos E, Lerner EP, Volak A, Cohen O, Hyman BT, Maguire CA, Vandenberghe LH.

Mol Ther Methods Clin Dev. 2018 Jul 23;10:197-209. doi: 10.1016/j.omtm.2018.07.006. eCollection 2018 Sep 21.

10.

Breaking and Sealing Barriers in Retinal Gene Therapy.

Miller JW, Vandenberghe LH.

Mol Ther. 2018 Sep 5;26(9):2081-2082. doi: 10.1016/j.ymthe.2018.08.003. Epub 2018 Aug 11. No abstract available.

PMID:
30107998
11.

Efficient Gene Transfer to Kidney Mesenchymal Cells Using a Synthetic Adeno-Associated Viral Vector.

Ikeda Y, Sun Z, Ru X, Vandenberghe LH, Humphreys BD.

J Am Soc Nephrol. 2018 Sep;29(9):2287-2297. doi: 10.1681/ASN.2018040426. Epub 2018 Jul 5.

PMID:
29976586
12.

The Assembly-Activating Protein Promotes Stability and Interactions between AAV's Viral Proteins to Nucleate Capsid Assembly.

Maurer AC, Pacouret S, Cepeda Diaz AK, Blake J, Andres-Mateos E, Vandenberghe LH.

Cell Rep. 2018 May 8;23(6):1817-1830. doi: 10.1016/j.celrep.2018.04.026.

13.

An Alternate Route for Adeno-associated Virus (AAV) Entry Independent of AAV Receptor.

Dudek AM, Pillay S, Puschnik AS, Nagamine CM, Cheng F, Qiu J, Carette JE, Vandenberghe LH.

J Virol. 2018 Mar 14;92(7). pii: e02213-17. doi: 10.1128/JVI.02213-17. Print 2018 Apr 1.

14.

Synthetic Adeno-Associated Viral Vector Efficiently Targets Mouse and Nonhuman Primate Retina In Vivo.

Carvalho LS, Xiao R, Wassmer SJ, Langsdorf A, Zinn E, Pacouret S, Shah S, Comander JI, Kim LA, Lim L, Vandenberghe LH.

Hum Gene Ther. 2018 Jul;29(7):771-784. doi: 10.1089/hum.2017.154. Epub 2018 Mar 20.

15.

Delivery of Adeno-Associated Virus Vectors in Adult Mammalian Inner-Ear Cell Subtypes Without Auditory Dysfunction.

Tao Y, Huang M, Shu Y, Ruprecht A, Wang H, Tang Y, Vandenberghe LH, Wang Q, Gao G, Kong WJ, Chen ZY.

Hum Gene Ther. 2018 Apr;29(4):492-506. doi: 10.1089/hum.2017.120. Epub 2018 Jan 22.

16.

Evaluating Efficiencies of Dual AAV Approaches for Retinal Targeting.

Carvalho LS, Turunen HT, Wassmer SJ, Luna-Velez MV, Xiao R, Bennett J, Vandenberghe LH.

Front Neurosci. 2017 Sep 8;11:503. doi: 10.3389/fnins.2017.00503. eCollection 2017.

17.

Single stranded adeno-associated virus achieves efficient gene transfer to anterior segment in the mouse eye.

Wang L, Xiao R, Andres-Mateos E, Vandenberghe LH.

PLoS One. 2017 Aug 1;12(8):e0182473. doi: 10.1371/journal.pone.0182473. eCollection 2017.

18.

Corrigendum: Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction.

Suzuki J, Hashimoto K, Xiao R, Vandenberghe LH, Liberman MC.

Sci Rep. 2017 May 22;7:46827. doi: 10.1038/srep46827.

19.

AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV Preparations.

Pacouret S, Bouzelha M, Shelke R, Andres-Mateos E, Xiao R, Maurer A, Mevel M, Turunen H, Barungi T, Penaud-Budloo M, Broucque F, Blouin V, Moullier P, Ayuso E, Vandenberghe LH.

Mol Ther. 2017 Jun 7;25(6):1375-1386. doi: 10.1016/j.ymthe.2017.04.001. Epub 2017 Apr 17.

20.

Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction.

Suzuki J, Hashimoto K, Xiao R, Vandenberghe LH, Liberman MC.

Sci Rep. 2017 Apr 3;7:45524. doi: 10.1038/srep45524. Erratum in: Sci Rep. 2017 May 22;7:46827.

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