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

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

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

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

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

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

scholarly journals 364. Neurological Correction of Mucopolysaccharidosis IIIB Mice by Haematopoietic Stem Cell Gene Therapy

2016 ◽  
Vol 24 ◽  
pp. S146
Author(s):  
Stuart M. Ellison ◽  
Rebecca Holley ◽  
Daniel Fil ◽  
John Mc Dermott ◽  
Nisha Senthivel ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Haematopoietic Stem Cell ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

scholarly journals Haematopoietic stem cell gene therapy with IL ‐1Ra rescues cognitive loss in mucopolysaccharidosis IIIA

2020 ◽  
Vol 12 (3) ◽  
Author(s):  
Helen Parker ◽  
Stuart M Ellison ◽  
Rebecca J Holley ◽  
Claire O'Leary ◽  
Aiyin Liao ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Haematopoietic Stem Cell ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy
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