Processing of iduronate 2-sulphatase in human fibroblasts
R Froissart, G Millat, M Mathieu, D Bozon… - Biochemical …, 1995 - portlandpress.com
Iduronate 2-sulphatase (IDS) is a lysosomal enzyme involved in degradation of dermatan
sulphate and heparan sulphate. Antigenic material was obtained either by purification of …
sulphate and heparan sulphate. Antigenic material was obtained either by purification of …
Molecular diagnosis of mucopolysaccharidosis type II (Hunter syndrome) by automated sequencing and computer-assisted interpretation: toward mutation mapping of …
JJ Jonsson, EL Aronovich, SE Braun… - American journal of …, 1995 - ncbi.nlm.nih.gov
Virtually all mutations causing Hunter syndrome (mucopolysaccharidosis type II) are
expected to be new mutations. Therefore, as a means of molecular diagnosis, we developed …
expected to be new mutations. Therefore, as a means of molecular diagnosis, we developed …
Metabolic correction of congenital erythropoietic porphyria by retrovirus-mediated gene transfer into Epstein-Barr virus-transformed B-cell lines
F Moreau-Gaudry, F Mazurier, M Bensidhoum, C Ged… - 1995 - ashpublications.org
Congenital erythropoietic porphyria (CEP) is an inherited metabolic disorder resulting from
the accumulation of porphyrins because of defective uroporphyrinogen III synthase …
the accumulation of porphyrins because of defective uroporphyrinogen III synthase …
Correction of deficient enzyme activity in a lysosomal storage disease, aspartylglucosaminuria, by enzyme replacement and retroviral gene transfer
N Enomaa, O Danos, L Peltonen, A Jalanko - Human gene therapy, 1995 - liebertpub.com
The ability of lysosomal enzymes to be secreted and subsequently captured by adjacent
cells provides an excellent basis for investigating different therapy strategies in lysosomal …
cells provides an excellent basis for investigating different therapy strategies in lysosomal …
Cellular gene therapy
JH Lee, HG Klein - Hematology/Oncology Clinics, 1995 - hemonc.theclinics.com
During the last decade, molecular genetic techniques have been used increasingly to
transfer human genes into mammalian cells, to correct and enhance cell function, and finally …
transfer human genes into mammalian cells, to correct and enhance cell function, and finally …
Mucopolysaccharidosis
O Danos, JM Heard - Molecular and Cell Biology of Human Gene …, 1995 - Springer
Most of the digestion process in the living cell takes place in the lysosome. These organelles
are formed in the trans Golgi network, from vesicles in which more than 40 different …
are formed in the trans Golgi network, from vesicles in which more than 40 different …
Gene therapy: clinical potential and relationships to drug treatment
KA Whartenby, AJ Marrogi, SM Freeman - Drugs, 1995 - Springer
Gene therapy has been defined as the alteration of the genetic material of a cell with
resultant benefit to a patient. Since its first clinical application in 1989, gene therapy has …
resultant benefit to a patient. Since its first clinical application in 1989, gene therapy has …