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Items: 1 to 50 of 282


Sub-2 Å Ewald curvature corrected structure of an AAV2 capsid variant.

Tan YZ, Aiyer S, Mietzsch M, Hull JA, McKenna R, Grieger J, Samulski RJ, Baker TS, Agbandje-McKenna M, Lyumkis D.

Nat Commun. 2018 Sep 7;9(1):3628. doi: 10.1038/s41467-018-06076-6.


An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs.

Sun J, Shao W, Chen X, Merricks EP, Wimsey L, Abajas YL, Niemeyer GP, Lothrop CD, Monahan PE, Samulski RJ, Nichols TC, Li C.

Mol Ther Methods Clin Dev. 2018 Aug 4;10:257-267. doi: 10.1016/j.omtm.2018.07.011. eCollection 2018 Sep 21.


Cryoprecipitate augments the global transduction of the adeno-associated virus serotype 9 after a systemic administration.

Chai Z, Zhang X, Rigsbee KM, Wang M, Samulski RJ, Li C.

J Control Release. 2018 Sep 28;286:415-424. doi: 10.1016/j.jconrel.2018.08.018. Epub 2018 Aug 11.


Nab Escaping AAV Mutants Isolated from Mouse Muscles.

Chai Z, Samulski RJ, Li C.

Bio Protoc. 2018 May 5;8(9). pii: e2841. doi: 10.21769/BioProtoc.2841.


Double-stranded RNA innate immune response activation from long-term adeno-associated virus vector transduction.

Shao W, Earley LF, Chai Z, Chen X, Sun J, He T, Deng M, Hirsch ML, Ting J, Samulski RJ, Li C.

JCI Insight. 2018 Jun 21;3(12). pii: 120474. doi: 10.1172/jci.insight.120474. [Epub ahead of print]


Impact of neutralizing antibodies against AAV is a key consideration in gene transfer to nonhuman primates.

Xiao W, Gao G, Ling C, Herzog RW, Xiao X, Samulski RJ.

Nat Med. 2018 Jun;24(6):699. doi: 10.1038/s41591-018-0062-2. No abstract available.


Blood-brain barrier shuttle peptides enhance AAV transduction in the brain after systemic administration.

Zhang X, He T, Chai Z, Samulski RJ, Li C.

Biomaterials. 2018 Sep;176:71-83. doi: 10.1016/j.biomaterials.2018.05.041. Epub 2018 May 26.


Efficient Capsid Antigen Presentation From Adeno-Associated Virus Empty Virions In Vivo.

Pei X, Earley LF, He Y, Chen X, Hall NE, Samulski RJ, Li C.

Front Immunol. 2018 Apr 19;9:844. doi: 10.3389/fimmu.2018.00844. eCollection 2018.


AAV6 K531 serves a dual function in selective receptor and antibody ADK6 recognition.

Bennett AD, Wong K, Lewis J, Tseng YS, Smith JK, Chipman P, McKenna R, Samulski RJ, Kleinschmidt J, Agbandje-McKenna M.

Virology. 2018 May;518:369-376. doi: 10.1016/j.virol.2018.03.007. Epub 2018 Mar 30.


AAV8 virions hijack serum proteins to increase hepatocyte binding for transduction enhancement.

Pei X, He T, Hall NE, Gerber D, Samulski RJ, Li C.

Virology. 2018 May;518:95-102. doi: 10.1016/j.virol.2018.02.007. Epub 2018 Feb 16.


Inhibition of antigen presentation during AAV gene therapy using virus peptides.

Shao W, Chen X, Samulski RJ, Hirsch ML, Li C.

Hum Mol Genet. 2018 Feb 15;27(4):601-613. doi: 10.1093/hmg/ddx427.


Protein Phosphatase Inhibitor-1 Gene Therapy in a Swine Model of Nonischemic Heart Failure.

Watanabe S, Ishikawa K, Fish K, Oh JG, Motloch LJ, Kohlbrenner E, Lee P, Xie C, Lee A, Liang L, Kho C, Leonardson L, McIntyre M, Wilson S, Samulski RJ, Kranias EG, Weber T, Akar FG, Hajjar RJ.

J Am Coll Cardiol. 2017 Oct 3;70(14):1744-1756. doi: 10.1016/j.jacc.2017.08.013.


Application of polyploid adeno-associated virus vectors for transduction enhancement and neutralizing antibody evasion.

Chai Z, Sun J, Rigsbee KM, Wang M, Samulski RJ, Li C.

J Control Release. 2017 Sep 28;262:348-356. doi: 10.1016/j.jconrel.2017.08.005. Epub 2017 Aug 5.


AAV Gene Therapy for Alcoholism: Inhibition of Mitochondrial Aldehyde Dehydrogenase Enzyme Expression in Hepatoma Cells.

Sanchez AC, Li C, Andrews B, Asenjo JA, Samulski RJ.

Hum Gene Ther. 2017 Sep;28(9):717-725. doi: 10.1089/hum.2017.043. Epub 2017 Jun 2.


AAV-Nrf2 Promotes Protection and Recovery in Animal Models of Oxidative Stress.

Liang KJ, Woodard KT, Weaver MA, Gaylor JP, Weiss ER, Samulski RJ.

Mol Ther. 2017 Mar 1;25(3):765-779. doi: 10.1016/j.ymthe.2016.12.016.


Systemic and Persistent Muscle Gene Expression in Rhesus Monkeys with a Liver De-Targeted Adeno-Associated Virus Vector.

Tarantal AF, Lee CCI, Martinez ML, Asokan A, Samulski RJ.

Hum Gene Ther. 2017 May;28(5):385-391. doi: 10.1089/hum.2016.130. Epub 2017 Jan 25.


β-cell-specific IL-35 therapy suppresses ongoing autoimmune diabetes in NOD mice.

Manzoor F, Johnson MC, Li C, Samulski RJ, Wang B, Tisch R.

Eur J Immunol. 2017 Jan;47(1):144-154. doi: 10.1002/eji.201646493. Epub 2016 Nov 25.


Direct interaction of human serum proteins with AAV virions to enhance AAV transduction: immediate impact on clinical applications.

Wang M, Sun J, Crosby A, Woodard K, Hirsch ML, Samulski RJ, Li C.

Gene Ther. 2017 Jan;24(1):49-59. doi: 10.1038/gt.2016.75. Epub 2016 Nov 11.


Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism.

Powell SK, Khan N, Parker CL, Samulski RJ, Matsushima G, Gray SJ, McCown TJ.

Gene Ther. 2016 Nov;23(11):807-814. doi: 10.1038/gt.2016.62. Epub 2016 Sep 15.


N-acetylaspartate supports the energetic demands of developmental myelination via oligodendroglial aspartoacylase.

Francis JS, Wojtas I, Markov V, Gray SJ, McCown TJ, Samulski RJ, Bilaniuk LT, Wang DJ, De Vivo DC, Janson CG, Leone P.

Neurobiol Dis. 2016 Dec;96:323-334. doi: 10.1016/j.nbd.2016.10.001. Epub 2016 Oct 4.


Retinal pigment epithelial cell expression of active Rap 1a by scAAV2 inhibits choroidal neovascularization.

Wang H, Han X, Bretz CA, Becker S, Gambhir D, Smith GW, Samulski RJ, Wittchen ES, Quilliam LA, Chrzanowska-Wodnicka M, Hartnett ME.

Mol Ther Methods Clin Dev. 2016 Aug 24;3:16056. doi: 10.1038/mtm.2016.56. eCollection 2016.


Identification and Validation of Small Molecules That Enhance Recombinant Adeno-associated Virus Transduction following High-Throughput Screens.

Nicolson SC, Li C, Hirsch ML, Setola V, Samulski RJ.

J Virol. 2016 Jul 27;90(16):7019-7031. doi: 10.1128/JVI.02953-15. Print 2016 Aug 15.


AAVR: A Multi-Serotype Receptor for AAV.

Summerford C, Johnson JS, Samulski RJ.

Mol Ther. 2016 Apr;24(4):663-6. doi: 10.1038/mt.2016.49. No abstract available. Erratum in: Mol Ther. 2016 Aug;24(8):1502.


The Adeno-Associated Virus Genome Packaging Puzzle.

Ling C, Wang Y, Lu Y, Wang L, Jayandharan GR, Aslanidi GV, Li B, Cheng B, Ma W, Lentz T, Ling C, Xiao X, Samulski RJ, Muzyczka N, Srivastava A.

J Mol Genet Med. 2015 Aug;9(3). pii: 175. Epub 2015 Jul 15. No abstract available.


Disruption of Microtubules Post-Virus Entry Enhances Adeno-Associated Virus Vector Transduction.

Xiao PJ, Mitchell AM, Huang L, Li C, Samulski RJ.

Hum Gene Ther. 2016 Apr;27(4):309-24. doi: 10.1089/hum.2016.008.


AAV Gene Therapy for MPS1-associated Corneal Blindness.

Vance M, Llanga T, Bennett W, Woodard K, Murlidharan G, Chungfat N, Asokan A, Gilger B, Kurtzberg J, Samulski RJ, Hirsch ML.

Sci Rep. 2016 Feb 22;6:22131. doi: 10.1038/srep22131.


Safety and biodistribution assessment of sc-rAAV2.5IL-1Ra administered via intra-articular injection in a mono-iodoacetate-induced osteoarthritis rat model.

Wang G, Evans CH, Benson JM, Hutt JA, Seagrave J, Wilder JA, Grieger JC, Samulski RJ, Terse PS.

Mol Ther Methods Clin Dev. 2016 Jan 13;3:15052. doi: 10.1038/mtm.2015.52. eCollection 2016.


Adeno-associated virus gene therapy vector scAAVIGF-I for transduction of equine articular chondrocytes and RNA-seq analysis.

Hemphill DD, McIlwraith CW, Slayden RA, Samulski RJ, Goodrich LR.

Osteoarthritis Cartilage. 2016 May;24(5):902-11. doi: 10.1016/j.joca.2015.12.001. Epub 2015 Dec 17.


Delivering Transgenic DNA Exceeding the Carrying Capacity of AAV Vectors.

Hirsch ML, Wolf SJ, Samulski RJ.

Methods Mol Biol. 2016;1382:21-39. doi: 10.1007/978-1-4939-3271-9_2.


Adeno-associated Virus as a Mammalian DNA Vector.

Salganik M, Hirsch ML, Samulski RJ.

Microbiol Spectr. 2015 Aug;3(4). doi: 10.1128/microbiolspec.MDNA3-0052-2014. Review.


Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer.

Li C, Wu S, Albright B, Hirsch M, Li W, Tseng YS, Agbandje-McKenna M, McPhee S, Asokan A, Samulski RJ.

Mol Ther. 2016 Feb;24(1):53-65. doi: 10.1038/mt.2015.134. Epub 2015 Jul 29.


Prediction of adeno-associated virus neutralizing antibody activity for clinical application.

Wang M, Crosby A, Hastie E, Samulski JJ, McPhee S, Joshua G, Samulski RJ, Li C.

Gene Ther. 2015 Dec;22(12):984-92. doi: 10.1038/gt.2015.69. Epub 2015 Jun 30.


Adeno-Associated Virus Vector Mediated Delivery of the HBV Genome Induces Chronic Hepatitis B Virus Infection and Liver Fibrosis in Mice.

Ye L, Yu H, Li C, Hirsch ML, Zhang L, Samulski RJ, Li W, Liu Z.

PLoS One. 2015 Jun 15;10(6):e0130052. doi: 10.1371/journal.pone.0130052. eCollection 2015.


scAAVIL-1ra dosing trial in a large animal model and validation of long-term expression with repeat administration for osteoarthritis therapy.

Goodrich LR, Grieger JC, Phillips JN, Khan N, Gray SJ, McIlwraith CW, Samulski RJ.

Gene Ther. 2015 Jul;22(7):536-45. doi: 10.1038/gt.2015.21. Epub 2015 Apr 23.


Adeno-associated virus at 50: a golden anniversary of discovery, research, and gene therapy success--a personal perspective.

Hastie E, Samulski RJ.

Hum Gene Ther. 2015 May;26(5):257-65. doi: 10.1089/hum.2015.025. Epub 2015 Apr 20. Review.


Recombinant adeno-associated virus vectors in the treatment of rare diseases.

Hastie E, Samulski RJ.

Expert Opin Orphan Drugs. 2015;3(6):675-689. Epub 2015 May 15.


Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial.

Monahan PE, Sun J, Gui T, Hu G, Hannah WB, Wichlan DG, Wu Z, Grieger JC, Li C, Suwanmanee T, Stafford DW, Booth CJ, Samulski JJ, Kafri T, McPhee SW, Samulski RJ.

Hum Gene Ther. 2015 Feb;26(2):69-81. doi: 10.1089/hum.2014.106. Epub 2015 Jan 21.


Intermediate filament protein accumulation in motor neurons derived from giant axonal neuropathy iPSCs rescued by restoration of gigaxonin.

Johnson-Kerner BL, Ahmad FS, Diaz AG, Greene JP, Gray SJ, Samulski RJ, Chung WK, Van Coster R, Maertens P, Noggle SA, Henderson CE, Wichterle H.

Hum Mol Genet. 2015 Mar 1;24(5):1420-31. doi: 10.1093/hmg/ddu556. Epub 2014 Nov 4.


AAV-Mediated Gene Therapy for Research and Therapeutic Purposes.

Samulski RJ, Muzyczka N.

Annu Rev Virol. 2014 Nov;1(1):427-51. doi: 10.1146/annurev-virology-031413-085355.


Enhanced transgene expression from recombinant single-stranded D-sequence-substituted adeno-associated virus vectors in human cell lines in vitro and in murine hepatocytes in vivo.

Ling C, Wang Y, Lu Y, Wang L, Jayandharan GR, Aslanidi GV, Li B, Cheng B, Ma W, Lentz T, Ling C, Xiao X, Samulski RJ, Muzyczka N, Srivastava A.

J Virol. 2015 Jan 15;89(2):952-61. doi: 10.1128/JVI.02581-14. Epub 2014 Oct 29.


Insight into the mechanism of inhibition of adeno-associated virus by the Mre11/Rad50/Nbs1 complex.

Lentz TB, Samulski RJ.

J Virol. 2015 Jan;89(1):181-94. doi: 10.1128/JVI.01990-14. Epub 2014 Oct 15.


Biology of adeno-associated viral vectors in the central nervous system.

Murlidharan G, Samulski RJ, Asokan A.

Front Mol Neurosci. 2014 Sep 19;7:76. doi: 10.3389/fnmol.2014.00076. eCollection 2014. Review.


Preclinical toxicity evaluation of AAV for pain: evidence from human AAV studies and from the pharmacology of analgesic drugs.

Pleticha J, Heilmann LF, Evans CH, Asokan A, Samulski RJ, Beutler AS.

Mol Pain. 2014 Sep 2;10:54. doi: 10.1186/1744-8069-10-54. Review.


Recombinant adeno-associated virus utilizes cell-specific infectious entry mechanisms.

Weinberg MS, Nicolson S, Bhatt AP, McLendon M, Li C, Samulski RJ.

J Virol. 2014 Nov;88(21):12472-84. doi: 10.1128/JVI.01971-14. Epub 2014 Aug 20.


Adeno-associated viral vectors show serotype specific transduction of equine joint tissue explants and cultured monolayers.

Hemphill DD, McIlwraith CW, Samulski RJ, Goodrich LR.

Sci Rep. 2014 Jul 29;4:5861. doi: 10.1038/srep05861.


Cardiac I-1c overexpression with reengineered AAV improves cardiac function in swine ischemic heart failure.

Ishikawa K, Fish KM, Tilemann L, Rapti K, Aguero J, Santos-Gallego CG, Lee A, Karakikes I, Xie C, Akar FG, Shimada YJ, Gwathmey JK, Asokan A, McPhee S, Samulski J, Samulski RJ, Sigg DC, Weber T, Kranias EG, Hajjar RJ.

Mol Ther. 2014 Dec;22(12):2038-45. doi: 10.1038/mt.2014.127. Epub 2014 Jul 15.


Human neural stem cells survive long term in the midbrain of dopamine-depleted monkeys after GDNF overexpression and project neurites toward an appropriate target.

Wakeman DR, Redmond DE Jr, Dodiya HB, Sladek JR Jr, Leranth C, Teng YD, Samulski RJ, Snyder EY.

Stem Cells Transl Med. 2014 Jun;3(6):692-701. doi: 10.5966/sctm.2013-0208. Epub 2014 Apr 17.

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