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Items: 13

1.

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.

Alterman JF, Godinho BMDC, Hassler MR, Ferguson CM, Echeverria D, Sapp E, Haraszti RA, Coles AH, Conroy F, Miller R, Roux L, Yan P, Knox EG, Turanov AA, King RM, Gernoux G, Mueller C, Gray-Edwards HL, Moser RP, Bishop NC, Jaber SM, Gounis MJ, Sena-Esteves M, Pai AA, DiFiglia M, Aronin N, Khvorova A.

Nat Biotechnol. 2019 Aug;37(8):884-894. doi: 10.1038/s41587-019-0205-0. Epub 2019 Aug 2.

PMID:
31375812
2.

Focal cooling of brain parenchyma in a transient large vessel occlusion model: proof-of-concept.

Caroff J, King RM, Mitchell JE, Marosfoi M, Licwinko JR, Gray-Edwards HL, Puri AS, Merrill TL, Gounis MJ.

J Neurointerv Surg. 2019 Jul 30. pii: neurintsurg-2019-015179. doi: 10.1136/neurintsurg-2019-015179. [Epub ahead of print]

PMID:
31363042
3.

Adeno-Associated Virus Gene Therapy in a Sheep Model of Tay-Sachs Disease.

Gray-Edwards HL, Randle AN, Maitland SA, Benatti HR, Hubbard SM, Canning PF, Vogel MB, Brunson BL, Hwang M, Ellis LE, Bradbury AM, Gentry AS, Taylor AR, Wooldridge AA, Wilhite DR, Winter RL, Whitlock BK, Johnson JA, Holland M, Salibi N, Beyers RJ, Sartin JL, Denney TS, Cox NR, Sena-Esteves M, Martin DR.

Hum Gene Ther. 2018 Mar;29(3):312-326. doi: 10.1089/hum.2017.163. Epub 2017 Sep 18.

PMID:
28922945
4.

Lipidomic Evaluation of Feline Neurologic Disease after AAV Gene Therapy.

Gray-Edwards HL, Jiang X, Randle AN, Taylor AR, Voss TL, Johnson AK, McCurdy VJ, Sena-Esteves M, Ory DS, Martin DR.

Mol Ther Methods Clin Dev. 2017 Jul 26;6:135-142. doi: 10.1016/j.omtm.2017.07.005. eCollection 2017 Sep 15.

5.

Novel Biomarkers of Human GM1 Gangliosidosis Reflect the Clinical Efficacy of Gene Therapy in a Feline Model.

Gray-Edwards HL, Regier DS, Shirley JL, Randle AN, Salibi N, Thomas SE, Latour YL, Johnston J, Golas G, Maguire AS, Taylor AR, Sorjonen DC, McCurdy VJ, Christopherson PW, Bradbury AM, Beyers RJ, Johnson AK, Brunson BL, Cox NR, Baker HJ, Denney TS, Sena-Esteves M, Tifft CJ, Martin DR.

Mol Ther. 2017 Apr 5;25(4):892-903. doi: 10.1016/j.ymthe.2017.01.009. Epub 2017 Feb 22.

6.

In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy.

Choudhury SR, Fitzpatrick Z, Harris AF, Maitland SA, Ferreira JS, Zhang Y, Ma S, Sharma RB, Gray-Edwards HL, Johnson JA, Johnson AK, Alonso LC, Punzo C, Wagner KR, Maguire CA, Kotin RM, Martin DR, Sena-Esteves M.

Mol Ther. 2016 Aug;24(7):1247-57. doi: 10.1038/mt.2016.84. Epub 2016 Apr 27.

7.

Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector.

Choudhury SR, Harris AF, Cabral DJ, Keeler AM, Sapp E, Ferreira JS, Gray-Edwards HL, Johnson JA, Johnson AK, Su Q, Stoica L, DiFiglia M, Aronin N, Martin DR, Gao G, Sena-Esteves M.

Mol Ther. 2016 Apr;24(4):726-35. doi: 10.1038/mt.2015.231. Epub 2015 Dec 28.

8.

Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy.

Gray-Edwards HL, Brunson BL, Holland M, Hespel AM, Bradbury AM, McCurdy VJ, Beadlescomb PM, Randle AN, Salibi N, Denney TS, Beyers RJ, Johnson AK, Voyles ML, Montgomery RD, Wilson DU, Hudson JA, Cox NR, Baker HJ, Sena-Esteves M, Martin DR.

Mol Genet Metab. 2015 Sep-Oct;116(1-2):80-7. doi: 10.1016/j.ymgme.2015.05.003. Epub 2015 May 8.

PMID:
25971245
9.

AAV-mediated gene delivery in a feline model of Sandhoff disease corrects lysosomal storage in the central nervous system.

Rockwell HE, McCurdy VJ, Eaton SC, Wilson DU, Johnson AK, Randle AN, Bradbury AM, Gray-Edwards HL, Baker HJ, Hudson JA, Cox NR, Sena-Esteves M, Seyfried TN, Martin DR.

ASN Neuro. 2015 Apr 13;7(2). pii: 1759091415569908. doi: 10.1177/1759091415569908. Print 2015 Mar-Apr.

10.

Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease.

McCurdy VJ, Rockwell HE, Arthur JR, Bradbury AM, Johnson AK, Randle AN, Brunson BL, Hwang M, Gray-Edwards HL, Morrison NE, Johnson JA, Baker HJ, Cox NR, Seyfried TN, Sena-Esteves M, Martin DR.

Gene Ther. 2015 Feb;22(2):181-9. doi: 10.1038/gt.2014.108. Epub 2014 Dec 4.

PMID:
25474439
11.

Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff disease.

Bradbury AM, Gray-Edwards HL, Shirley JL, McCurdy VJ, Colaco AN, Randle AN, Christopherson PW, Bird AC, Johnson AK, Wilson DU, Hudson JA, De Pompa NL, Sorjonen DC, Brunson BL, Jeyakumar M, Platt FM, Baker HJ, Cox NR, Sena-Esteves M, Martin DR.

Exp Neurol. 2015 Jan;263:102-12. doi: 10.1016/j.expneurol.2014.09.020. Epub 2014 Oct 5.

12.

Sustained normalization of neurological disease after intracranial gene therapy in a feline model.

McCurdy VJ, Johnson AK, Gray-Edwards HL, Randle AN, Brunson BL, Morrison NE, Salibi N, Johnson JA, Hwang M, Beyers RJ, Leroy SG, Maitland S, Denney TS, Cox NR, Baker HJ, Sena-Esteves M, Martin DR.

Sci Transl Med. 2014 Apr 9;6(231):231ra48. doi: 10.1126/scitranslmed.3007733.

13.

High resolution MRI anatomy of the cat brain at 3 Tesla.

Gray-Edwards HL, Salibi N, Josephson EM, Hudson JA, Cox NR, Randle AN, McCurdy VJ, Bradbury AM, Wilson DU, Beyers RJ, Denney TS, Martin DR.

J Neurosci Methods. 2014 Apr 30;227:10-7. doi: 10.1016/j.jneumeth.2014.01.035. Epub 2014 Feb 10.

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