Delivering Hematopoietic Stem Cell Gene Therapy Treatments for Neurological Lysosomal Diseases | Zendy

Rebecca Holley | Zendy; Shaun Wood | Zendy; Brian Bigger | Zendy

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Delivering Hematopoietic Stem Cell Gene Therapy Treatments for Neurological Lysosomal Diseases

Author(s) -

Rebecca Holley,

Shaun Wood,

Brian Bigger

Publication year - 2018

Publication title -

acs chemical neuroscience

Language(s) - Uncategorized

Resource type - Journals

SCImago Journal Rank - 1.158

H-Index - 69

ISSN - 1948-7193

DOI - 10.1021/acschemneuro.8b00408

Subject(s) - genetic enhancement , enzyme replacement therapy , stem cell , haematopoiesis , hematopoietic stem cell transplantation , hematopoietic stem cell , lysosomal storage disease , ex vivo , stem cell therapy , transplantation , disease , medicine , cell therapy , biology , cancer research , bioinformatics , immunology , gene , in vivo , pathology , microbiology and biotechnology , genetics

Neurological lysosomal storage diseases are rare, inherited conditions resulting mainly from lysosomal enzyme deficiencies. Current treatments, such as enzyme replacement therapy and hematopoietic stem cell transplantation, fail to effectively treat neurological disease due to insufficient brain delivery of the missing enzyme. Ex vivo gene therapy approaches to overexpress the missing enzyme in hematopoietic stem cells prior to transplant are an emerging technology that has the potential to offer a viable therapy for patients with these debilitating diseases.

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