20. Development of Lentiviral Mediated Stem Cell Gene Therapy for Mucopolysaccharidosis Type IIIA

Molecular Therapy ◽

10.1016/s1525-0016(16)35033-x ◽

2014 ◽

Vol 22 ◽

pp. S8

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Mucopolysaccharidosis Type ◽

Type Iiia ◽

Cell Gene ◽

Mucopolysaccharidosis Type Iiia ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Lentiviral mediated stem cell gene therapy corrects a mouse model of mucopolysaccharidosis type IIIA

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2010.11.026 ◽

2011 ◽

Vol 102 (2) ◽

pp. S8

Author(s):

Brian Bigger ◽

Alex Langford-Smith ◽

Fiona Wilkinson ◽

Kia Langford-Smith ◽

Ana Sergijenko ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Mouse Model ◽

Mucopolysaccharidosis Type ◽

Type Iiia ◽

Cell Gene ◽

Mucopolysaccharidosis Type Iiia ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Pre-clinical workup of lentiviral mediated stem cell gene therapy for mucopolysaccharidosis type IIIA

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2014.12.076 ◽

2015 ◽

Vol 114 (2) ◽

pp. S41

Author(s):

Stuart M. Ellison ◽

Ana Sergijenko ◽

Alex Langford-Smith ◽

Kia Langford-Smith ◽

Fiona Wilkinson ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Mucopolysaccharidosis Type ◽

Type Iiia ◽

Cell Gene ◽

Mucopolysaccharidosis Type Iiia ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Case report of the first patient treated with ex-vivo autologous haematopoietic stem cell gene therapy transplant in mucopolysaccharidosis type IIIA

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2019.11.225 ◽

2020 ◽

Vol 129 (2) ◽

pp. S90 ◽

Author(s):

Jane Louise Kinsella ◽

Heather Church ◽

Wendy Ogden ◽

Ceri Jones ◽

Karen Buckland ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Case Report ◽

Ex Vivo ◽

Haematopoietic Stem Cell ◽

Mucopolysaccharidosis Type ◽

Cell Gene ◽

Mucopolysaccharidosis Type Iiia ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Ex-vivo autologous stem cell gene therapy clinical trial for mucopolysaccharidosis type IIIA: Update on phase I/II clinical trial

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2020.12.126 ◽

2021 ◽

Vol 132 (2) ◽

pp. S56-S57

Author(s):

Jane Louise Kinsella ◽

Simon Jones ◽

Adrian J. Thrasher ◽

Claire Booth ◽

Karen F. Buckland ◽

...

Keyword(s):

Clinical Trial ◽

Gene Therapy ◽

Stem Cell ◽

Ex Vivo ◽

Mucopolysaccharidosis Type ◽

Cell Gene ◽

Mucopolysaccharidosis Type Iiia ◽

Stem Cell Gene ◽

Therapy Clinical Trial ◽

Stem Cell Gene Therapy

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Brain targeted stem cell gene therapy provides long-term correction of mucopolysaccharidosis type II

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2018.12.059 ◽

2019 ◽

Vol 126 (2) ◽

pp. S31-S32

Author(s):

Brian Bigger ◽

Ai-Yin Liao ◽

Helene Gleitz ◽

Rebecca Holley

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Mucopolysaccharidosis Type ◽

Type Ii ◽

Mucopolysaccharidosis Type Ii ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Hematopoietic Stem Cell Gene Therapy Corrects Neuropathic Phenotype in Murine Model of Mucopolysaccharidosis Type II

Human Gene Therapy ◽

10.1089/hum.2014.158 ◽

2015 ◽

Vol 26 (6) ◽

pp. 357-366 ◽

Author(s):

Taichi Wakabayashi ◽

Yohta Shimada ◽

Kazumasa Akiyama ◽

Takashi Higuchi ◽

Takahiro Fukuda ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Murine Model ◽

Mucopolysaccharidosis Type ◽

Type Ii ◽

Mucopolysaccharidosis Type Ii ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Brain‐targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms

EMBO Molecular Medicine ◽

10.15252/emmm.201708730 ◽

2018 ◽

Vol 10 (7) ◽

Author(s):

Hélène FE Gleitz ◽

Ai Yin Liao ◽

James R Cook ◽

Samuel F Rowlston ◽

Gabriella MA Forte ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Mucopolysaccharidosis Type ◽

Type Ii ◽

Mucopolysaccharidosis Type Ii ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Neurological correction of mucopolysaccharidosis type IIIB mice by haematopoietic stem cell gene therapy

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2016.11.043 ◽

2017 ◽

Vol 120 (1-2) ◽

pp. S28

Author(s):

Brian Bigger ◽

Stuart Ellison ◽

Daniel Fil ◽

Claire O'leary ◽

John McDermott ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Haematopoietic Stem Cell ◽

Mucopolysaccharidosis Type ◽

Type Iiib ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy ◽

Mucopolysaccharidosis Type Iiib

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Whole body correction of severe mucopolysaccharidosis type II by lentiviral-mediated stem cell gene therapy with blood-brain barrier-crossing peptides

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2016.11.124 ◽

2017 ◽

Vol 120 (1-2) ◽

pp. S56-S57 ◽

Author(s):

Hélène F.E. Gleitz ◽

Claire O'Leary ◽

Rebecca J. Holley ◽

Brian W. Bigger

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Blood Brain Barrier ◽

Mucopolysaccharidosis Type ◽

Type Ii ◽

Mucopolysaccharidosis Type Ii ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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PS1220 EX-VIVO HEMATOPOIETIC STEM CELL GENE THERAPY (GT) FOR MUCOPOLYSACCHARIDOSIS TYPE I HURLER (MPSIH): PRELIMINARY RESULTS FROM A PHASE I/II CLINICAL STUDY

10.1097/01.hs9.0000563164.44448.4c ◽

2019 ◽

Vol 3 (S1) ◽

pp. 556-557

Author(s):

M.E. Bernardo ◽

B. Gentner ◽

F. Tucci ◽

F. Fumagalli ◽

P. Silvani ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Ex Vivo ◽

Type I ◽

Mucopolysaccharidosis Type ◽

Mucopolysaccharidosis Type I ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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