Lysosomal storage

1. Tripeptidyl peptidase 1 decreases lysosomal storage in mice.

   “Importantly, IV administration of TPP1 with the peptide significantly reduces brain lysosomal storage, increases lifespan and improves neurological function.”

   Animalsin LINCL micerecombinant TPP1 coadministered with a 36-residue Apolipoprotein E-derived peptidebrain

   Effective intravenous therapy for neurodegenerative disease with a therapeutic enzyme and a peptide that mediates delivery to the brain

   Molecular therapy : the journal of the American Society of Gene Therapy·26 Dec 2013

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1. Syngeneic bone marrow transplantation decreases lysosomal storage in mice.

   “Spleen, liver, cornea, and glomerular mesangial cells showed essentially complete correction at all radiation doses.”

   Animalsmurine MPS VII (gusmps/gusmps)

   Increased life span and correction of metabolic defects in murine mucopolysaccharidosis type VII after syngeneic bone marrow transplantation

   Blood·1 Dec 1991

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