Treatment of lysosomal storage disorders: focus on the neuronal ceroid-lipofuscinoses
Recent advances in our understanding of lysosomal storage disorders (LSDs) may lead to
new therapies to treat the neuronal ceroid-lipofuscinoses (NCLs). In this review, enzyme
replacement therapy, gene therapy, cell-mediated therapy and pharmaceutical treatments
are considered across the LSDs and extended to therapies for the NCLs. It is likely that a
combination of approaches will produce the most beneficial clinical outcome for treatment of
pathologies displayed by the NCLs.
new therapies to treat the neuronal ceroid-lipofuscinoses (NCLs). In this review, enzyme
replacement therapy, gene therapy, cell-mediated therapy and pharmaceutical treatments
are considered across the LSDs and extended to therapies for the NCLs. It is likely that a
combination of approaches will produce the most beneficial clinical outcome for treatment of
pathologies displayed by the NCLs.
Recent advances in our understanding of lysosomal storage disorders (LSDs) may lead to new therapies to treat the neuronal ceroid-lipofuscinoses (NCLs). In this review, enzyme replacement therapy, gene therapy, cell-mediated therapy and pharmaceutical treatments are considered across the LSDs and extended to therapies for the NCLs. It is likely that a combination of approaches will produce the most beneficial clinical outcome for treatment of pathologies displayed by the NCLs.
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