Bone marrow transplantation for lysosomal storage disorders
نویسنده
چکیده
منابع مشابه
Specific Treatment for Lysosomal Storage Disorders: Enzyme Replacement Therapy, Bone Marrow Transplant and Others
Received December 15, 2003 Abstract In this article, we review specific therapies that tackle the basic biochemical defects of lysosomal storage diseases. These include bone marrow transplantation, substrate deprivation therapy, enzyme replacement therapy and enzyme enhancement therapy. We particularly update the progress of development of enzyme replacement therapy, which plays a major role in...
متن کاملMurine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation.
We demonstrated previously that short term administration of recombinant beta-glucuronidase to newborn mice with mucopolysaccharidosis type VII reduced lysosomal storage in many tissues. Lysosomal storage accumulated gradually after cessation of enzyme replacement therapy. Mice alive at 1 yr of age had decreased bone deformities and less lysosomal storage in cortical neurons. Here we compare th...
متن کاملReview of Effect of Storage Time before Freezing on Stem Cells Surveillance, Collected from Cord Blood, Peripheral Blood and Bone Marrow
Introduction: The use of cord stem cells, as a cell source for bone marrow reconstruction in patient under hematopoietic stem cell transplantation is enhancing. The amount of CD34+cells collected from cord blood compared to peripheral blood and bone marrow are less, but their collectionprocesure is easier. Delay in freezing has negative effect on cells surveillance and reduce their ability in ...
متن کاملIntracerebral transplantation of mesenchymal stem cells into acid sphingomyelinase-deficient mice delays the onset of neurological abnormalities and extends their life span.
Types A and B Niemann-Pick disease (NPD) are lysosomal storage disorders resulting from loss of acid sphingomyelinase (ASM) activity. We have used a knockout mouse model of NPD (ASMKO mice) to evaluate the effects of direct intracerebral transplantation of bone marrow-derived mesenchymal stem cells (MSCs) on the progression of neurological disease in this disorder. MSCs were transduced with a r...
متن کاملBone marrow transplantation for treatment of lysosomal storage diseases. Proceedings of a colloquium. May 7, 1985, Washington, D.C.
Will reading habit influence your life? Many say yes. Reading bone marrow transplantation for treatment of lysosomal storage diseases proceedings of a colloquium held may 7 1985 washington dc is a good habit; you can develop this habit to be such interesting way. Yeah, reading habit will not only make you have any favourite activity. It will be one of guidance of your life. When reading has bec...
متن کامل