Examination of a blood-brain barrier targeting β-galactosidase-monoclonal antibody fusion protein in a murine model of GM1-gangliosidosis

Author:

Przybilla Michael J.,Stewart Christine,Carlson Timothy W.,Ou Li,Koniar Brenda L.,Sidhu Rohini,Kell Pamela J.,Jiang Xuntian,Jarnes Jeanine R.,O'Sullivan M. Gerard,Whitley Chester B.

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology

Reference35 articles.

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2. Infantile gangliosidoses: mapping a timeline of clinical changes;Jarnes Utz;Mol. Genet. Metab.,2017

3. The juvenile gangliosidoses: A timeline of clinical change;King;Mol. Genet. Metab. Rep.,2020

4. Enzyme replacement for lysosomal diseases;Brady;Annu. Rev. Med.,2006

5. Enzyme replacement therapy for lysosomal diseases: lessons from 20 years of experience and remaining challenges;Desnick;Annu. Rev. Genomics Hum. Genet.,2012

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