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Steven J. Gray - Publications

Affiliations: 
2008-2018 Gene Therapy Center University of North Carolina, Chapel Hill, Chapel Hill, NC 
 2017- Neurology and Pediatrics University of Texas Southwestern Medical Center, Dallas, TX, United States 
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101 high-probability publications. We are testing a new system for linking publications to authors. You can help! If you notice any inaccuracies, please sign in and mark papers as correct or incorrect matches. If you identify any major omissions or other inaccuracies in the publication list, please let us know.

Year Citation  Score
2023 Ryckman AE, Deschenes NM, Quinville BM, Osmon KJL, Mitchell M, Chen Z, Gray SJ, Walia JS. Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study. Molecular Therapy. Methods & Clinical Development. 32: 101168. PMID 38205442 DOI: 10.1016/j.omtm.2023.101168  0.3
2023 Gumusgoz E, Kasiri S, Verma M, Wu J, Villarreal Acha D, Marriam U, Fyffe-Maricich S, Lin A, Chen X, Gray SJ, Minassian BA. CSTB gene replacement improves neuroinflammation, neurodegeneration and ataxia in murine type 1 progressive myoclonus epilepsy. Gene Therapy. PMID 38135787 DOI: 10.1038/s41434-023-00433-x  0.352
2023 Murray SJ, Wellby MP, Barrell GK, Russell KN, Deane AR, Wynyard JR, Gray SJ, Palmer DN, Mitchell NL. Efficacy of dual intracerebroventricular and intravitreal gene therapy in sheep prompts the first clinical trial to treat CLN5 Batten disease. Frontiers in Pharmacology. 14: 1212235. PMID 37942487 DOI: 10.3389/fphar.2023.1212235  0.309
2023 Vyas M, Deschenes NM, Osmon KJL, Chen Z, Ahmad I, Kot S, Thompson P, Richmond C, Gray SJ, Walia JS. Efficacy of Adeno-Associated Virus Serotype 9-Mediated Gene Therapy for AB-Variant GM2 Gangliosidosis. International Journal of Molecular Sciences. 24. PMID 37834060 DOI: 10.3390/ijms241914611  0.4
2023 Ling Q, Herstine JA, Bradbury A, Gray SJ. AAV-based in vivo gene therapy for neurological disorders. Nature Reviews. Drug Discovery. PMID 37658167 DOI: 10.1038/s41573-023-00766-7  0.351
2023 Casy W, Garza IT, Chen X, Dong T, Hu Y, Kanchwala M, Trygg CB, Shyng C, Xing C, Bunnell BA, Braun SE, Gray SJ. SMRT Sequencing Enables High-Throughput Identification of Novel AAVs from Capsid Shuffling and Directed Evolution. Genes. 14. PMID 37628711 DOI: 10.3390/genes14081660  0.737
2023 Mitchell NL, Murray SJ, Wellby MP, Barrell GK, Russell KN, Deane AR, Wynyard JR, Palmer MJ, Pulickan A, Prendergast PM, Casy W, Gray SJ, Palmer DN. Long-term safety and dose escalation of intracerebroventricular CLN5 gene therapy in sheep supports clinical translation for CLN5 Batten disease. Frontiers in Genetics. 14: 1212228. PMID 37614821 DOI: 10.3389/fgene.2023.1212228  0.307
2023 Wong H, Hooper AW, Kang HR, Lee SJ, Zhao J, Sadhu C, Rawat S, Gray SJ, Hampson DR. CNS-dominant human FMRP isoform rescues seizures, fear, and sleep abnormalities in Fmr1-KO mice. Jci Insight. 8. PMID 37288657 DOI: 10.1172/jci.insight.169650  0.338
2023 Chen X, Lim DA, Lawlor MW, Dimmock D, Vite C, Lester T, Tavakkoli F, Sadhu C, Prasad S, Gray SJ. Biodistribution of adeno-associated virus gene therapy following CSF-directed administration. Human Gene Therapy. PMID 36606687 DOI: 10.1089/hum.2022.163  0.378
2022 Chen X, Dong T, Hu Y, Shaffo FC, Belur NR, Mazzulli JR, Gray SJ. AAV9/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 disease. The Journal of Clinical Investigation. PMID 35025759 DOI: 10.1172/JCI146286  0.302
2021 Ling Q, Rioux M, Hu Y, Lee M, Gray SJ. Adeno-associated viral vector serotype 9-based gene replacement therapy for -related Leigh syndrome. Molecular Therapy. Methods & Clinical Development. 23: 158-168. PMID 34703839 DOI: 10.1016/j.omtm.2021.09.001  0.318
2021 Judson MC, Shyng C, Simon JM, Davis CR, Punt AM, Salmon MT, Miller NW, Ritola KD, Elgersma Y, Amaral DG, Gray SJ, Philpot BD. Dual-isoform hUBE3A gene transfer improves behavioral and seizure outcomes in Angelman syndrome model mice. Jci Insight. 6. PMID 34676830 DOI: 10.1172/jci.insight.144712  0.739
2021 Kot S, Karumuthil-Melethil S, Woodley E, Zaric V, Thompson P, Chen Z, Lykken E, Keimel JG, Kaemmerer WF, Gray SJ, Walia JS. Investigating Immune Responses to the scAAV9- Gene Therapy Treatment in Tay-Sachs Disease and Sandhoff Disease Mouse Models. International Journal of Molecular Sciences. 22. PMID 34201771 DOI: 10.3390/ijms22136751  0.323
2021 Armao D, Bouldin TW, Bailey RM, Gray SJ. Extensive rod and cone photoreceptor-cell degeneration in rat models of giant axonal neuropathy: implications for gene therapy of human disease. Ophthalmic Genetics. 1-4. PMID 33955818 DOI: 10.1080/13816810.2021.1923036  0.311
2021 Sinnett SE, Boyle E, Lyons C, Gray SJ. Engineered microRNA-based regulatory element permits safe high-dose miniMECP2 gene therapy in Rett mice. Brain : a Journal of Neurology. PMID 33950254 DOI: 10.1093/brain/awab182  0.351
2021 Presa M, Bailey RM, Davis C, Murphy T, Cook J, Walls R, Wilpan H, Bogdanik L, Lenk GM, Burgess RW, Gray SJ, Lutz C. AAV9-mediated FIG4 delivery prolongs life span in Charcot Marie Tooth disease type 4J mouse model. The Journal of Clinical Investigation. PMID 33878035 DOI: 10.1172/JCI137159  0.326
2021 Francis JS, Markov V, Wojtas ID, Gray S, McCown T, Samulski RJ, Figueroa M, Leone P. Preclinical biodistribution, tropism, and efficacy of oligotropic AAV/Olig001 in a mouse model of congenital white matter disease. Molecular Therapy. Methods & Clinical Development. 20: 520-534. PMID 33614826 DOI: 10.1016/j.omtm.2021.01.009  0.329
2020 Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN, Boye SL, Boye SE, George LA, Salabarria S, Corti M, Byrne BJ, Tremblay JP. Current Clinical Applications of in vivo Gene Therapy with AAVs. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 33309881 DOI: 10.1016/j.ymthe.2020.12.007  0.357
2020 Bradbury AM, Bagel JH, Nguyen D, Lykken EA, Pesayco Salvador J, Jiang X, Swain GP, Assenmacher CA, Hendricks IJ, Miyadera K, Hess RS, Ostrager A, ODonnell P, Sands MS, Ory DS, ... ... Gray SJ, et al. Krabbe disease successfully treated via monotherapy of intrathecal gene therapy. The Journal of Clinical Investigation. PMID 32773406 DOI: 10.1172/Jci133953  0.613
2020 Fagiolini M, Patrizi A, LeBlanc J, Jin LW, Maezawa I, Sinnett S, Gray SJ, Molholm S, Foxe JJ, Johnston MV, Naidu S, Blue M, Hossain A, Kadam S, Zhao X, et al. Intellectual and Developmental Disabilities Research Centers: a multidisciplinary approach to understand the pathogenesis of MECP2-related disorders. Neuroscience. PMID 32360592 DOI: 10.1016/J.Neuroscience.2020.04.037  0.312
2020 Bailey RM, Rozenberg A, Gray SJ. Comparison of High-Dose Intracisterna Magna and Lumbar Puncture Intrathecal Delivery of AAV9 in Mice to Treat Neuropathies. Brain Research. 146832. PMID 32289279 DOI: 10.1016/J.Brainres.2020.146832  0.41
2020 Lykken EA, Rozenberg AJ, Gray SJ. Combination intrathecal and intravenous gene therapy reveals a dominant role for treatment age in determining survival and behavioral outcomes in the mouse model of infantile neuronal ceroid lipofuscinosis Molecular Genetics and Metabolism. 129: S104. DOI: 10.1016/J.Ymgme.2019.11.265  0.365
2020 Chen X, Shaffo F, Dong T, Hu Y, Belur NR, Mazzulli JR, Gray SJ. Preclinical efficacy and safety evaluation of scAAV9/CLN7 gene replacement therapy in rodents Molecular Genetics and Metabolism. 129: S40. DOI: 10.1016/J.Ymgme.2019.11.079  0.346
2019 Armao D, Bouldin TW, Bailey RM, Hooper JE, Bharucha DX, Gray SJ. Advancing the pathologic phenotype of giant axonal neuropathy: early involvement of the ocular lens. Orphanet Journal of Rare Diseases. 14: 27. PMID 30709364 DOI: 10.1186/S13023-018-0957-5  0.389
2019 Woodley E, Osmon KJL, Thompson P, Richmond C, Chen Z, Gray SJ, Walia JS. Efficacy of a Bicistronic Vector for Correction of Sandhoff Disease in a Mouse Model. Molecular Therapy. Methods & Clinical Development. 12: 47-57. PMID 30534578 DOI: 10.1016/J.Omtm.2018.10.011  0.417
2019 Lutz C, Presa M, Davis C, Cook J, Murphy T, Burgess R, Bailey RM, Gray SJ. Preclinical gene therapy in a mouse model of Charcot-Marie-Tooth disease type 4J Molecular Genetics and Metabolism. 126: S96-S97. DOI: 10.1016/J.Ymgme.2018.12.241  0.455
2019 Gray SJ, Rozenberg A, Lykken E, Padegimas L, Miller T. Intrathecal and intravenous combination gene therapy in the mouse model of infantile neuronal ceroid lipofuscinosis extends lifespan and improves behavioral outcomes in moderately affected mice Molecular Genetics and Metabolism. 126: S63-S64. DOI: 10.1016/J.Ymgme.2018.12.149  0.386
2019 Chen X, Snanoudj-Verber S, Pollard L, Cathey S, Gray SJ. Therapeutic efficacy and safety of scAAV9/AGA gene therapy in aspartylglucosaminuria mice Molecular Genetics and Metabolism. 126: S38. DOI: 10.1016/J.Ymgme.2018.12.078  0.382
2019 Bailey RM, Presa M, Lutz C, Gray SJ. Development of scAAV9/SUMF1 gene therapy for multiple sulfatase deficiency Molecular Genetics and Metabolism. 126: S27. DOI: 10.1016/J.Ymgme.2018.12.045  0.395
2018 Mitchell NL, Russell KN, Wellby MP, Wicky HE, Schoderboeck L, Barrell GK, Melzer TR, Gray SJ, Hughes SM, Palmer DN. Longitudinal In Vivo Monitoring of the CNS Demonstrates the Efficacy of Gene Therapy in a Sheep Model of CLN5 Batten Disease. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 30078766 DOI: 10.1016/J.Ymthe.2018.07.015  0.458
2018 Ramsingh AI, Gray SJ, Reilly A, Koday M, Bratt D, Koday MT, Murnane R, Smedley J, Hu Y, Messer A, Fuller DH. Sustained AAV9-mediated expression of a non-self protein in the CNS of non-human primates after immunomodulation. Plos One. 13: e0198154. PMID 29874260 DOI: 10.1371/Journal.Pone.0198154  0.385
2018 Lykken EA, Shyng C, Edwards RJ, Rozenberg A, Gray SJ. Recent progress and considerations for AAV gene therapies targeting the central nervous system. Journal of Neurodevelopmental Disorders. 10: 16. PMID 29776328 DOI: 10.1186/S11689-018-9234-0  0.76
2018 Bailey RM, Armao D, Nagabhushan Kalburgi S, Gray SJ. Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy. Molecular Therapy. Methods & Clinical Development. 9: 160-171. PMID 29766026 DOI: 10.1016/J.Omtm.2018.02.005  0.433
2018 Marshall MS, Issa Y, Jakubauskas B, Stoskute M, Elackattu V, Marshall JN, Bogue W, Nguyen D, Hauck Z, Rue E, Karumuthil-Melethil S, Zaric V, Bosland M, van Breemen RB, Givogri MI, ... Gray SJ, et al. Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe's Disease after Global Gene Therapy. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 29433937 DOI: 10.1016/J.Ymthe.2018.01.009  0.449
2018 Rozenberg AJ, Lykken E, Spratt K, Miller TJ, Gray SJ. Combination dosing of CLN1 gene therapy extends lifespan in a mouse model of infantile neuronal ceroid lipofuscinosis Molecular Genetics and Metabolism. 123: S124. DOI: 10.1016/J.Ymgme.2017.12.338  0.365
2018 Davis A, Lykken E, Sharma A, Padegimas L, Kerns S, Gray S. AAV vector comparability across mammalian and insect cell production platforms for treatment of lysosomal diseases Molecular Genetics and Metabolism. 123: S37. DOI: 10.1016/J.Ymgme.2017.12.076  0.339
2017 Sinnett SE, Gray SJ. Recent endeavors in MECP2 gene transfer for gene therapy of Rett syndrome. Discovery Medicine. 24: 153-159. PMID 29272692  0.381
2017 Mandel RJ, Marmion DJ, Kirik D, Chu Y, Heindel C, McCown T, Gray SJ, Kordower JH. Novel oligodendroglial alpha synuclein viral vector models of multiple system atrophy: studies in rodents and nonhuman primates. Acta Neuropathologica Communications. 5: 47. PMID 28619074 DOI: 10.1186/S40478-017-0451-7  0.35
2017 Gadalla KKE, Vudhironarit T, Hector RD, Sinnett S, Bahey NG, Bailey MES, Gray SJ, Cobb SR. Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome. Molecular Therapy. Methods & Clinical Development. 5: 180-190. PMID 28497075 DOI: 10.1016/J.Omtm.2017.04.007  0.455
2017 Sinnett SE, Hector RD, Gadalla KKE, Heindel C, Chen D, Zaric V, Bailey MES, Cobb SR, Gray SJ. Improved MECP2 Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery. Molecular Therapy. Methods & Clinical Development. 5: 106-115. PMID 28497072 DOI: 10.1016/J.Omtm.2017.04.006  0.425
2017 Banning A, König JF, Gray SJ, Tikkanen R. Functional Analysis of the Ser149/Thr149 Variants of Human Aspartylglucosaminidase and Optimization of the Coding Sequence for Protein Production. International Journal of Molecular Sciences. 18. PMID 28346360 DOI: 10.3390/Ijms18040706  0.376
2017 Rozenberg AJ, Gray SJ. Early intrathecal gene therapy extends lifespan and improves quality of life in a mouse model for infantile neuronal ceroid lipofuscinosis Molecular Genetics and Metabolism. 120: S117. DOI: 10.1016/J.Ymgme.2016.11.301  0.355
2016 Banning A, Gülec C, Rouvinen J, Gray SJ, Tikkanen R. Identification of Small Molecule Compounds for Pharmacological Chaperone Therapy of Aspartylglucosaminuria. Scientific Reports. 6: 37583. PMID 27876883 DOI: 10.1038/Srep37583  0.317
2016 Francis JS, Wojtas I, Markov V, Gray SJ, McCown TJ, Samulski RJ, Bilaniuk LT, Wang DJ, De Vivo DC, Janson CG, Leone P. N-acetylaspartate supports the energetic demands of developmental myelination via oligodendroglial aspartoacylase. Neurobiology of Disease. PMID 27717881 DOI: 10.1016/J.Nbd.2016.10.001  0.344
2016 Karumuthil-Melethil S, Gray SJ. Immunological considerations for treating globoid cell leukodystrophy. Journal of Neuroscience Research. 94: 1349-58. PMID 27638617 DOI: 10.1002/Jnr.23874  0.384
2016 Karumuthil-Melethil S, Marshall MS, Heindel C, Jakubauskas B, Bongarzone ER, Gray SJ. Intrathecal administration of AAV/GALC vectors in 10-11-day-old twitcher mice improves survival and is enhanced by bone marrow transplant. Journal of Neuroscience Research. 94: 1138-51. PMID 27638599 DOI: 10.1002/Jnr.23882  0.432
2016 Powell SK, Khan N, Parker CL, Samulski RJ, Matsushima G, Gray SJ, McCown TJ. Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism. Gene Therapy. PMID 27628693 DOI: 10.1038/Gt.2016.62  0.438
2016 Bongarzone ER, Escolar ML, Gray SJ, Kafri T, Vite CH, Sands MS. Insights into the Pathogenesis and Treatment of Krabbe Disease. Pediatric Endocrinology Reviews : Per. 13: 689-96. PMID 27491217  0.525
2016 Armao D, Bailey RM, Bouldin TW, Kim Y, Gray SJ. Autonomic nervous system involvement in the giant axonal neuropathy (GAN) KO mouse: implications for human disease. Clinical Autonomic Research : Official Journal of the Clinical Autonomic Research Society. PMID 27369358 DOI: 10.1007/S10286-016-0365-7  0.301
2016 Gray SJ. Timing of Gene Therapy Interventions: The Earlier, the Better. Molecular Therapy : the Journal of the American Society of Gene Therapy. 24: 1017-8. PMID 27324445 DOI: 10.1038/Mt.2016.20  0.43
2016 Osmon KJ, Woodley E, Thompson P, Ong K, Karumuthil-Melethil S, Keimel JG, Mark BL, Mahuran D, Gray S, Walia JS. Systemic Gene Transfer of a Hexosaminidase Variant Using a scAAV9.47 Vector Corrects GM2 Gangliosidosis in Sandhoff Mice. Human Gene Therapy. PMID 27199088 DOI: 10.1089/Hum.2016.015  0.408
2016 Karumuthil-Melethil S, Nagabhushan Kalburgi S, Thompson P, Tropak M, Kaytor MD, Keimel JG, Mark BL, Mahuran D, Walia JS, Gray SJ. NOVEL VECTOR DESIGN AND HEXOSAMINIDASE VARIANT ENABLING SELF-COMPLEMENTARY AAV FOR THE TREATMENT OF TAY-SACHS DISEASE. Human Gene Therapy. PMID 27197548 DOI: 10.1089/Hum.2016.013  0.429
2016 Tropak MB, Yonekawa S, Karumuthil-Melethil S, Thompson P, Wakarchuk W, Gray SJ, Walia JS, Mark BL, Mahuran D. Construction of a hybrid β-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in vivo. Molecular Therapy. Methods & Clinical Development. 3: 15057. PMID 26966698 DOI: 10.1038/Mtm.2015.57  0.344
2016 Katz DM, Bird A, Coenraads M, Gray SJ, Menon DU, Philpot BD, Tarquinio DC. Rett Syndrome: Crossing the Threshold to Clinical Translation. Trends in Neurosciences. 39: 100-13. PMID 26830113 DOI: 10.1016/J.Tins.2015.12.008  0.582
2016 Thompson P, Osmon K, Woodley E, Karumuthil-Melethil S, Gray SJ, Walia JS. 725. Assessment of CSF Route for Gene Delivery in Sandhoff Mice Using AAV9 Expressing an Hexosaminidase Isoenzyme Molecular Therapy. 24: S286. DOI: 10.1016/S1525-0016(16)33533-X  0.371
2016 Gurda BL, Swain G, Bagel JH, Prociuk M, Gray SJ, Fitzgerald C, O'Donnell P, Vite CH. 611. Promoter Evaluation of AAV Gene Therapy in the Central Nervous System for Feline Niemann-Pick Type C Disease Molecular Therapy. 24: S242. DOI: 10.1016/S1525-0016(16)33419-0  0.423
2016 Bailey RM, Rozenberg A, Toppins C, Gray SJ. 604. Comparison of Intra-Cisterna Magna and Lumbar Puncture Intrathecal Delivery of scAAV9 GeneTherapy for Giant Axonal Neuropathy Molecular Therapy. 24: S239. DOI: 10.1016/S1525-0016(16)33412-8  0.369
2016 Woodley EJ, Osmon K, Thompson P, Karumuthil-Melethil S, Gray S, Walia J. 551. Enhancement of Gene Therapy Treatment for Sandhoff Disease Through Complimentary Drug Therapy Molecular Therapy. 24: S221. DOI: 10.1016/S1525-0016(16)33359-7  0.456
2016 Osmon KJ, Woodley E, Thompson P, Karumuthil-Melethil S, Gray S, Walia J. 363. Improvement of Sandhoff Phenotype Following Intravenous Injection of Adeno-Associated Viral Vector Expressing a Hexosaminidase Isoenzyme in Adult Sandhoff Mice: Preclinical Safety and Efficacy Study Molecular Therapy. 24: S145-S146. DOI: 10.1016/S1525-0016(16)33172-0  0.371
2016 Rozenberg AJ, Kalburgi SN, Gray SJ. 362. Translatable Gene Therapy for Infantile Neuronal Lipofuscinosis Molecular Therapy. 24: S145. DOI: 10.1016/S1525-0016(16)33171-9  0.44
2016 Karumuthil-Melethil S, Marshall M, Bongarzone ER, Gray SJ. 57. Intrathecal Administration of AAV/GALC Vectors in Juvenile Twitcher Mice Improves Survival and Is Enhanced by Bone Marrow Transplant Molecular Therapy. 24: S25. DOI: 10.1016/S1525-0016(16)32866-0  0.442
2016 Li Y, Shea LK, Jiang X, Gray SJ, Ory DS, Sands MS. Combination therapy increases lifespan and improves clinicobehavioral performance in the murine model of globoid cell leukodystrophy Molecular Genetics and Metabolism. 117: S73. DOI: 10.1016/J.Ymgme.2015.12.338  0.568
2015 Hawkins-Salsbury JA, Shea L, Jiang X, Hunter DA, Guzman AM, Reddy AS, Qin EY, Li Y, Gray SJ, Ory DS, Sands MS. Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophy. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 35: 6495-505. PMID 25904800 DOI: 10.1523/Jneurosci.4199-14.2015  0.62
2015 Goodrich LR, Grieger JC, Phillips JN, Khan N, Gray SJ, McIlwraith CW, Samulski RJ. scAAVIL-1ra dosing trial in a large animal model and validation of long-term expression with repeat administration for osteoarthritis therapy. Gene Therapy. 22: 536-45. PMID 25902762 DOI: 10.1038/Gt.2015.21  0.361
2015 Ekins S, Litterman NK, Arnold RJ, Burgess RW, Freundlich JS, Gray SJ, Higgins JJ, Langley B, Willis DE, Notterpek L, Pleasure D, Sereda MW, Moore A. A brief review of recent Charcot-Marie-Tooth research and priorities. F1000research. 4: 53. PMID 25901280 DOI: 10.12688/F1000Research.6160.1  0.335
2015 Powell SK, Rivera-Soto R, Gray SJ. Viral expression cassette elements to enhance transgene target specificity and expression in gene therapy. Discovery Medicine. 19: 49-57. PMID 25636961  0.31
2015 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. Intermediate filament protein accumulation in motor neurons derived from giant axonal neuropathy iPSCs rescued by restoration of gigaxonin. Human Molecular Genetics. 24: 1420-31. PMID 25398950 DOI: 10.1093/Hmg/Ddu556  0.402
2015 Osmon KJ, Woodley E, Thompson P, Ong K, Karumuthil-Melethil S, Mark B, Mahuran D, Gray SJ, Walia JS. MG-110 Intravenous neonatal gene therapy corrects GM2 gangliosidoses in sandhoff mice for ‘long-term’, by using an aav expressing a new hexosaminidase variant Journal of Medical Genetics. 52: A4.2-A4. DOI: 10.1136/Jmedgenet-2015-103578.10  0.377
2015 Sinnett SE, Kalburgi SN, Gray SJ. 713. IntraCSF Administration of AAV9/MeCP2 Extends Lifespan of MeCP2-Null Mice While Preventing Toxicity Associated With IV Administration Molecular Therapy. 23: S284-S285. DOI: 10.1016/S1525-0016(16)34322-2  0.428
2015 Karumuthil-Melethil S, Thompson P, Walia JS, Mark B, Mahuran D, Gray SJ. 712. Improved Reduction in GM2 Ganglioside Accumulation in Tay-Sachs Mice Using a New Hexosaminidase Variant Molecular Therapy. 23: S284. DOI: 10.1016/S1525-0016(16)34321-0  0.391
2015 Osmon KJ, Woodley E, Thompson P, Ong K, Karumuthil-Melethil S, Mark B, Mahuran D, Gray SJ, Walia JS. 711. Intravenous Neonatal Gene Therapy Corrects GM2 Gangliosidosis in Sandhoff Mice for Long-Term, By Using AAV Viral Vector Expressing a New Hexosaminidase Variant Molecular Therapy. 23: S283-S284. DOI: 10.1016/S1525-0016(16)34320-9  0.421
2015 Powell SK, Khan N, Parker C, Jude Samulski R, Matsushima G, Gray SJ, McCown TJ. 306. Characterization of a Novel Adeno-Associated Viral Vector with Preferential Oligodendrocyte Tropism Molecular Therapy. 23: S123-S124. DOI: 10.1016/S1525-0016(16)33915-6  0.395
2015 Bailey RM, Armao D, Nagabhushan Kalburgi S, Gray SJ. 15. Development of Intrathecal scAAV9 Gene Therapy for Giant Axonal Neuropathy Molecular Therapy. 23: S6-S7. DOI: 10.1016/S1525-0016(16)33619-X  0.427
2014 Kantor B, Bailey RM, Wimberly K, Kalburgi SN, Gray SJ. Methods for gene transfer to the central nervous system. Advances in Genetics. 87: 125-97. PMID 25311922 DOI: 10.1016/B978-0-12-800149-3.00003-2  0.426
2014 Kantor B, McCown T, Leone P, Gray SJ. Clinical applications involving CNS gene transfer. Advances in Genetics. 87: 71-124. PMID 25311921 DOI: 10.1016/B978-0-12-800149-3.00002-0  0.471
2013 Simonato M, Bennett J, Boulis NM, Castro MG, Fink DJ, Goins WF, Gray SJ, Lowenstein PR, Vandenberghe LH, Wilson TJ, Wolfe JH, Glorioso JC. Progress in gene therapy for neurological disorders. Nature Reviews. Neurology. 9: 277-91. PMID 23609618 DOI: 10.1038/Nrneurol.2013.56  0.437
2013 Sinici I, Yonekawa S, Tkachyova I, Gray SJ, Samulski RJ, Wakarchuk W, Mark BL, Mahuran DJ. In cellulo examination of a beta-alpha hybrid construct of beta-hexosaminidase A subunits, reported to interact with the GM2 activator protein and hydrolyze GM2 ganglioside. Plos One. 8: e57908. PMID 23483939 DOI: 10.1371/Journal.Pone.0057908  0.371
2013 Nagabhushan Kalburgi S, Khan NN, Gray SJ. Recent gene therapy advancements for neurological diseases. Discovery Medicine. 15: 111-9. PMID 23449113  0.336
2013 Goodrich LR, Phillips JN, McIlwraith CW, Foti SB, Grieger JC, Gray SJ, Samulski RJ. Optimization of scAAVIL-1ra In Vitro and In Vivo to Deliver High Levels of Therapeutic Protein for Treatment of Osteoarthritis. Molecular Therapy. Nucleic Acids. 2: e70. PMID 23385523 DOI: 10.1038/Mtna.2012.61  0.347
2013 Mussche S, Devreese B, Nagabhushan Kalburgi S, Bachaboina L, Fox JC, Shih HJ, Van Coster R, Samulski RJ, Gray SJ. Restoration of cytoskeleton homeostasis after gigaxonin gene transfer for giant axonal neuropathy. Human Gene Therapy. 24: 209-19. PMID 23316953 DOI: 10.1089/Hum.2012.107  0.424
2013 Gray SJ, Nagabhushan Kalburgi S, McCown TJ, Jude Samulski R. Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates. Gene Therapy. 20: 450-9. PMID 23303281 DOI: 10.1038/Gt.2012.101  0.377
2013 Gadalla KK, Bailey ME, Spike RC, Ross PD, Woodard KT, Kalburgi SN, Bachaboina L, Deng JV, West AE, Samulski RJ, Gray SJ, Cobb SR. Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 18-30. PMID 23011033 DOI: 10.1038/Mt.2012.200  0.434
2013 Gray SJ. Gene therapy and neurodevelopmental disorders Neuropharmacology. 68: 136-142. PMID 22750077 DOI: 10.1016/J.Neuropharm.2012.06.024  0.432
2013 Sloniowski SP, Fox JC, Gray SJ. Perspectives in using gene therapy for lysosomal storage diseases Drugs of the Future. 38: 635-643. DOI: 10.1358/Dof.2013.038.09.2038787  0.451
2012 Bowles DE, McPhee SW, Li C, Gray SJ, Samulski JJ, Camp AS, Li J, Wang B, Monahan PE, Rabinowitz JE, Grieger JC, Govindasamy L, Agbandje-McKenna M, Xiao X, Samulski RJ. Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector. Molecular Therapy : the Journal of the American Society of Gene Therapy. 20: 443-55. PMID 22068425 DOI: 10.1038/Mt.2011.237  0.768
2012 Lentz TB, Gray SJ, Samulski RJ. Viral vectors for gene delivery to the central nervous system. Neurobiology of Disease. 48: 179-88. PMID 22001604 DOI: 10.1016/J.Nbd.2011.09.014  0.47
2012 Federici T, Taub JS, Baum GR, Gray SJ, Grieger JC, Matthews KA, Handy CR, Passini MA, Samulski RJ, Boulis NM. Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs. Gene Therapy. 19: 852-9. PMID 21918551 DOI: 10.1038/Gt.2011.130  0.379
2011 Gray SJ, Choi VW, Asokan A, Haberman RA, McCown TJ, Samulski RJ. Production of recombinant adeno-associated viral vectors and use in in vitro and in vivo administration. Current Protocols in Neuroscience / Editorial Board, Jacqueline N. Crawley ... [Et Al.]. Unit 4.17. PMID 21971848 DOI: 10.1002/0471142301.Ns0417S57  0.7
2011 Li C, Xiao P, Gray SJ, Weinberg MS, Samulski RJ. Combination therapy utilizing shRNA knockdown and an optimized resistant transgene for rescue of diseases caused by misfolded proteins. Proceedings of the National Academy of Sciences of the United States of America. 108: 14258-63. PMID 21844342 DOI: 10.1073/Pnas.1109522108  0.411
2011 Gray SJ, Matagne V, Bachaboina L, Yadav S, Ojeda SR, Samulski RJ. Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 1058-69. PMID 21487395 DOI: 10.1038/Mt.2011.72  0.355
2011 Gray SJ, Foti SB, Schwartz JW, Bachaboina L, Taylor-Blake B, Coleman J, Ehlers MD, Zylka MJ, McCown TJ, Samulski RJ. Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors. Human Gene Therapy. 22: 1143-53. PMID 21476867 DOI: 10.1089/Hum.2010.245  0.372
2011 Snyder BR, Gray SJ, Quach ET, Huang JW, Leung CH, Samulski RJ, Boulis NM, Federici T. Comparison of adeno-associated viral vector serotypes for spinal cord and motor neuron gene delivery. Human Gene Therapy. 22: 1129-35. PMID 21443428 DOI: 10.1089/Hum.2011.008  0.328
2010 Gray SJ, Blake BL, Criswell HE, Nicolson SC, Samulski RJ, McCown TJ. Erratum: Corrigendum to "Directed Evolution of a Novel Adeno-associated Virus (AAV) Vector That Crosses the Seizure-compromised Blood-Brain Barrier (BBB)" (Molecular Therapy (2010) 18 (570-578) 10.1038/mt.2009.292) Molecular Therapy. 18. PMID 28178554 DOI: 10.1038/Mt.2010.6  0.342
2010 Gray SJ, Woodard KT, Samulski RJ. Viral vectors and delivery strategies for CNS gene therapy. Therapeutic Delivery. 1: 517-34. PMID 22833965 DOI: 10.4155/Tde.10.50  0.444
2010 Mendell JR, Campbell K, Rodino-Klapac L, Sahenk Z, Shilling C, Lewis S, Bowles D, Gray S, Li C, Galloway G, Malik V, Coley B, Clark KR, Li J, Xiao X, et al. Dystrophin immunity in Duchenne's muscular dystrophy. The New England Journal of Medicine. 363: 1429-37. PMID 20925545 DOI: 10.1056/Nejmoa1000228  0.749
2010 Hollis ER, Jamshidi P, Lorenzana AO, Lee JK, Gray SJ, Samulski RJ, Zheng B, Tuszynski MH. Transient demyelination increases the efficiency of retrograde AAV transduction. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 1496-500. PMID 20502445 DOI: 10.1038/Mt.2010.97  0.32
2010 Gray SJ, Blake BL, Criswell HE, Nicolson SC, Samulski RJ, McCown TJ, Li W. Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB). Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 570-8. PMID 20040913 DOI: 10.1038/Mt.2009.292  0.329
2009 Hewitt FC, Li C, Gray SJ, Cockrell S, Washburn M, Samulski RJ. Reducing the risk of adeno-associated virus (AAV) vector mobilization with AAV type 5 vectors. Journal of Virology. 83: 3919-29. PMID 19211760 DOI: 10.1128/Jvi.02466-08  0.399
2008 Gray SJ, Samulski RJ. Optimizing gene delivery vectors for the treatment of heart disease. Expert Opinion On Biological Therapy. 8: 911-22. PMID 18549322 DOI: 10.1517/14712598.8.7.911  0.404
2007 Gray SJ, Gerhardt J, Doerfler W, Small LE, Fanning E. An origin of DNA replication in the promoter region of the human fragile X mental retardation (FMR1) gene. Molecular and Cellular Biology. 27: 426-37. PMID 17101793 DOI: 10.1128/Mcb.01382-06  0.64
2007 Gray SJ, Liu G, Altman AL, Small LE, Fanning E. Discrete functional elements required for initiation activity of the Chinese hamster dihydrofolate reductase origin beta at ectopic chromosomal sites. Experimental Cell Research. 313: 109-20. PMID 17078947 DOI: 10.1016/J.Yexcr.2006.09.020  0.709
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