Literature DB >> 804420

Investigations in enzyme replacement therapy in lipid storage diseases.

R O Brady, P G Pentchev, A G Gal.   

Abstract

Enzyme replacement appears to offer much promise as specific therapeutic procedures for patients with Fabry's disease and Gaucher's disease. However, enzyme replacement in patients with Tay-Sachs disease and other heritable metabolic disorders where the central nervous system is affected will require first the development of effective methods for the delivery of exogenous enzymes to the brain; such methods are not yet available.

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Year:  1975        PMID: 804420

Source DB:  PubMed          Journal:  Fed Proc        ISSN: 0014-9446


  8 in total

Review 1.  Biochemistry and genetics of gangliosidoses.

Authors:  K Sandhoff; H Christomanou
Journal:  Hum Genet       Date:  1979       Impact factor: 4.132

Review 2.  Basic findings and current developments in sphingolipidoses.

Authors:  H Pilz; R Heipertz; D Seidel
Journal:  Hum Genet       Date:  1979-03-12       Impact factor: 4.132

Review 3.  Gaucher disease as a paradigm of current issues regarding single gene mutations of humans.

Authors:  E Beutler
Journal:  Proc Natl Acad Sci U S A       Date:  1993-06-15       Impact factor: 11.205

4.  Immunological aspects of urate oxidase therapy in hyperuricaemia.

Authors:  D A Fitzpatrick; O FitzGerald; K F McGeeney
Journal:  Ir J Med Sci       Date:  1977-06       Impact factor: 1.568

5.  Evidence for specific recognition sites mediating clearance of lysosomal enzymes in vivo.

Authors:  P Stahl; H Six; J S Rodman; P Schlesinger; D R Tulsiani; O Touster
Journal:  Proc Natl Acad Sci U S A       Date:  1976-11       Impact factor: 11.205

6.  Clearance of human N-acetyl-beta-hexosaminidases from rat circulation.

Authors:  T Bearpark; J L Stirling
Journal:  Biochem J       Date:  1977-12-15       Impact factor: 3.857

7.  Studies in vivo of the tissue uptake, cellular distribution and catabolic turnover of exogenous glucocerebrosidase in rat.

Authors:  S Morrone; P G Pentchev; J Baynes; S Thorpe
Journal:  Biochem J       Date:  1981-03-15       Impact factor: 3.857

8.  Investigations on therapeutic glucocerebrosidases through paired detection with fluorescent activity-based probes.

Authors:  Wouter W Kallemeijn; Saskia Scheij; Sascha Hoogendoorn; Martin D Witte; Daniela Herrera Moro Chao; Cindy P A A van Roomen; Roelof Ottenhoff; Herman S Overkleeft; Rolf G Boot; Johannes M F G Aerts
Journal:  PLoS One       Date:  2017-02-16       Impact factor: 3.240

  8 in total

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