Gene therapy for lysosomal storage disorders.
NS Yew, SH Cheng - Current Opinion in Molecular Therapeutics, 2001 - europepmc.org
NS Yew, SH Cheng
Current Opinion in Molecular Therapeutics, 2001•europepmc.orgDeficiencies in one or several of the numerous degradative enzymes that reside in the
lysosome often result in one of many clinically severe diseases, almost all of which have no
currently available therapy. Although bone marrow transplantation, enzyme replacement
and substrate inhibition therapies are being considered, gene therapy represents an
increasingly attractive approach, particularly for those lysosomal storage diseases with
neurological manifestations. This review summarizes the most recent advances in …
lysosome often result in one of many clinically severe diseases, almost all of which have no
currently available therapy. Although bone marrow transplantation, enzyme replacement
and substrate inhibition therapies are being considered, gene therapy represents an
increasingly attractive approach, particularly for those lysosomal storage diseases with
neurological manifestations. This review summarizes the most recent advances in …
Deficiencies in one or several of the numerous degradative enzymes that reside in the lysosome often result in one of many clinically severe diseases, almost all of which have no currently available therapy. Although bone marrow transplantation, enzyme replacement and substrate inhibition therapies are being considered, gene therapy represents an increasingly attractive approach, particularly for those lysosomal storage diseases with neurological manifestations. This review summarizes the most recent advances in developing gene therapies for this large and heterogeneous group of disorders.
europepmc.org