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Rachel M. Bailey

university of texas system

H指数:19

被引数:2,449

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AAV-mediated gene therapy in a model of SLC13A5 citrate transporter disorder rescues epileptic and metabolic phenotypes

AAV介导的基因疗法在SLC13A5柠檬酸转运蛋白障碍模型中挽救了癫痫和代谢表型

Journal of Clinical Investigation

IF13.6

2026-04-150

OA

T2 Hyperintensities in Gracile Tracts of Cervical Spinal Cord in Giant Axonal Neuropathy (GAN)

颈脊髓 gracile 束的 T2 高信号在巨大轴索神经病 (GAN) 中的表现

IF3.1

2025-08-160

OAAI

Nitrous oxide enhances MR-guided focused ultrasound delivery of gene therapy to the murine hippocampus

一氧化二氮增强MR引导聚焦超声向小鼠海马体递送基因治疗

IF4.5

2025-05-060

PRE

Preclinical use of a clinically-relevant scAAV9/SUMF1 vector for the treatment of multiple sulfatase deficiency

COMMUNICATIONS MEDICINE

IF6.3

2025-01-270

OA

Neurological, metabolic and inflammatory phenotypes in a mouse model of ECHS1 deficiency

ECHS1缺乏小鼠模型中的神经病学、代谢和炎症表型

BRAIN COMMUNICATIONS

IF4.5

2025-01-010

OAAI

Developmental phenotype and quality of life in SLC13A5 citrate transporter disorder

DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

IF4.3

2024-12-220

PRE

Intrathecal Gene Therapy for Giant Axonal Neuropathy

NEW ENGLAND JOURNAL OF MEDICINE

IF78.5

2024-03-219

PRE

Bharucha-Goebel, Diana X.; Todd, Joshua J.; Saade, Dimah; Norato, Gina; Jain, Minal; Lehky, Tanya; Bailey, Rachel M.; Chichester, Jessica A.; Calcedo, Roberto; Armao, Diane; Foley, A. Reghan; Mohassel, Payam; Tesfaye, Eshetu; Carlin, Bradley P.; Seremula, Beth; Waite, Melissa; Zein, Wadih M.; Huryn, Laryssa A.; Crawford, Thomas O.; Sumner, Charlotte J.; Hoke, Ahmet; Heiss, John D.; Charnas, Lawrence; Hooper, Jody E.; Bouldin, Thomas W.; Kang, Elizabeth M.; Rybin, Denis; Gray, Steven J.; Bonnemann, Carsten G.

Efficacy of a scAAV9/SUMF1 viral vector for the treatment of multiple sulfatase deficiency

MOLECULAR GENETICS AND METABOLISM

IF3.5

2022-02-010

PRE

AAV9-mediated FIG4 delivery prolongs life span in Charcot-Marie-Tooth disease type 4J mouse model

JOURNAL OF CLINICAL INVESTIGATION

IF13.6

2021-06-0120

OA

Preclinical studies to support the intrathecal delivery of scAAV9/SUMF1 as a gene replacement therapy for multiple sulfatase deficiency

MOLECULAR GENETICS AND METABOLISM

IF3.5

2021-02-012

PREAI

Preclinical gene therapy in a mouse model of Charcot-Marie-Tooth disease type 4J

MOLECULAR GENETICS AND METABOLISM

IF3.5

2019-02-010

PREAI

Development of scAAV9/SUMF1 gene therapy for multiple sulfatase deficiency

MOLECULAR GENETICS AND METABOLISM

IF3.5

2019-02-010

PREAI

Advancing the pathologic phenotype of giant axonal neuropathy: early involvement of the ocular lens

ORPHANET JOURNAL OF RARE DISEASES

IF3.5

2019-02-0110

OAAI

Autonomic nervous system involvement in the giant axonal neuropathy (GAN) KO mouse: implications for human disease (vol 26, pg 307, 2016)

CLINICAL AUTONOMIC RESEARCH

IF3.4

2016-08-110

PREAI

Autonomic nervous system involvement in the giant axonal neuropathy (GAN) KO mouse: implications for human disease

CLINICAL AUTONOMIC RESEARCH

IF3.4

2016-07-0111

OAAI

Comparison of Intra-Cisterna Magna and Lumbar Puncture Intrathecal Delivery of scAAV9 GeneTherapy for Giant Axonal Neuropathy

MOLECULAR THERAPY

IF12

2016-05-010

OAAI

Development of Intrathecal scAAV9 Gene Therapy for Giant Axonal Neuropathy

MOLECULAR THERAPY

IF12

2015-05-012

OAAI

Effects of the C57BL/6 strain background on tauopathy progression in the rTg4510 mouse model

MOLECULAR NEURODEGENERATION

IF17.5

2014-01-1526

OAAI

LRRK2 phosphorylates novel tau epitopes and promotes tauopathy

ACTA NEUROPATHOLOGICA

IF9.3

2013-10-1190

OAAI

In vivo functional brain mapping in a conditional mouse model of human tauopathy (taup301l) reveals reduced neural activity in memory formation structures

MOLECULAR NEURODEGENERATION

IF17.5

2013-02-0438

OAAI