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Bigger, Brian W
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1

Design and validation of a GMP stem cell manufacturing prot..:

Ellison, Stuart ; Buckland, Karen ; Learmonth, Yuko...
Molecular Therapy - Methods & Clinical Development.  32 (2024)  2 - p. 101271 , 2024
Link: https://doi.org/10.1016/..
 
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2

Systemic immune challenge exacerbates neurodegeneration in ..:

Mandolfo, Oriana ; Parker, Helen ; Aguado, Èlia...
EMBO Molecular Medicine.  16 (2024)  7 - p. 1579-1602 , 2024
Link: https://doi.org/10.1038/..
 
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3

Improving newborn screening test performance for metachroma..:

WU, Teresa H.Y. ; Brown, Heather A. ; Church, Heather J....
Molecular Genetics and Metabolism.  142 (2024)  1 - p. 108349 , 2024
Link: https://doi.org/10.1016/..
 
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4

Phenotypic characterisation of the Mucopolysaccharidosis Ty..:

Andreou, Tereza ; Ishikawa-Learmonth, Yuko ; Bigger, Brian W
Molecular Genetics and Metabolism.  139 (2023)  4 - p. 107651 , 2023
Link: https://doi.org/10.1016/..
 
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5

Sustained long-term disease correction in a murine model of..:

Ellison, Stuart ; Liao, Aiyin ; Gleitz, Hélène F.E....
Molecular Therapy - Methods & Clinical Development.  31 (2023)  - p. 101127 , 2023
Link: https://doi.org/10.1016/..
 
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6

Intraparenchymal convection enhanced delivery of AAV in she..:

O'Leary, Claire ; Forte, Gabriella ; Mitchell, Nadia L....
Journal of Translational Medicine.  21 (2023)  1 - p. , 2023
Link: https://doi.org/10.1186/..
 
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7

Clinical trial update: Ex-vivo autologous hematopoietic ste..:

Jones, Simon A. ; Kinsella, Jane L. ; Potter, Jane...
Molecular Genetics and Metabolism.  135 (2022)  2 - p. S63-S64 , 2022
Link: https://doi.org/10.1016/..
 
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8

Current and Future Treatment of Mucopolysaccharidosis (MPS)..:

Horgan, Claire ; Jones, Simon A. ; Bigger, Brian W..
International Journal of Molecular Sciences.  23 (2022)  9 - p. 4854 , 2022
Link: https://doi.org/10.3390/..
 
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9

Delivering gene therapy for mucopolysaccharide diseases:

Wood, Shaun R. ; Bigger, Brian W.
Frontiers in Molecular Biosciences.  9 (2022)  - p. , 2022
Link: https://doi.org/10.3389/..
 
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10

Bile acid biosynthesis in Smith-Lemli-Opitz syndrome bypass..:

Abdel-Khalik, Jonas ; Hearn, Thomas ; Dickson, Alison L....
The Journal of Steroid Biochemistry and Molecular Biology.  206 (2021)  - p. 105794 , 2021
Link: https://doi.org/10.1016/..
 
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11

Enzyme replacement therapy and hematopoietic stem cell tran..:

Potter, Jane E. ; Petts, Gemma ; Ghosh, Arunabha...
Orphanet Journal of Rare Diseases.  16 (2021)  1 - p. , 2021
Link: https://doi.org/10.1186/..
 
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12

Ex-vivo autologous stem cell gene therapy clinical trial fo..:

Kinsella, Jane Louise ; Jones, Simon ; Thrasher, Adrian J....
Molecular Genetics and Metabolism.  132 (2021)  2 - p. S56-S57 , 2021
Link: https://doi.org/10.1016/..
 
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13

High dose genistein in Sanfilippo syndrome: A randomised co..:

Ghosh, Arunabha ; Rust, Stewart ; Langford‐Smith, Kia...
Journal of Inherited Metabolic Disease.  44 (2021)  5 - p. 1248-1262 , 2021
Link: https://doi.org/10.1002/..
 
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14

Dual Purpose Vectors for Rare Neurological Diseases:

Bigger, Brian W.
Molecular Therapy.  28 (2020)  10 - p. 2104-2105 , 2020
Link: https://doi.org/10.1016/..
 
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15

Haematopoietic stem cell gene therapy withIL‐1Ra rescues co..:

Parker, Helen ; Ellison, Stuart M ; Holley, Rebecca J...
EMBO Molecular Medicine.  12 (2020)  3 - p. , 2020
Link: https://doi.org/10.15252..
 
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