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Current clinical applications of in vivo gene therapy with AAVs
Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …
Lysosomes as dynamic regulators of cell and organismal homeostasis
Exciting new discoveries have transformed the view of the lysosome from a static organelle
dedicated to the disposal and recycling of cellular waste to a highly dynamic structure that …
dedicated to the disposal and recycling of cellular waste to a highly dynamic structure that …
Past, present, and future perspectives of transcription factor EB (TFEB): mechanisms of regulation and association with disease
Transcription factor EB (TFEB), a member of the MiT/TFE family of basic helix-loop-helix
leucine zipper transcription factors, is an established central regulator of the …
leucine zipper transcription factors, is an established central regulator of the …
Lysosomes as a therapeutic target
SR Bonam, F Wang, S Muller - Nature reviews Drug discovery, 2019 - nature.com
Lysosomes are membrane-bound organelles with roles in processes involved in degrading
and recycling cellular waste, cellular signalling and energy metabolism. Defects in genes …
and recycling cellular waste, cellular signalling and energy metabolism. Defects in genes …
Lysosomal storage diseases
FM Platt, A d'Azzo, BL Davidson, EF Neufeld… - Nature reviews Disease …, 2018 - nature.com
Lysosomal storage diseases (LSDs) are a group of over 70 diseases that are characterized
by lysosomal dysfunction, most of which are inherited as autosomal recessive traits. These …
by lysosomal dysfunction, most of which are inherited as autosomal recessive traits. These …
Principles and functions of metabolic compartmentalization
Metabolism has historically been studied at the levels of whole cells, whole tissues and
whole organisms. As a result, our understanding of how compartmentalization—the spatial …
whole organisms. As a result, our understanding of how compartmentalization—the spatial …
Mechanisms and functions of lysosome positioning
Lysosomes have been classically considered terminal degradative organelles, but in recent
years they have been found to participate in many other cellular processes, including killing …
years they have been found to participate in many other cellular processes, including killing …
The rapidly evolving view of lysosomal storage diseases
Lysosomal storage diseases are a group of metabolic disorders caused by deficiencies of
several components of lysosomal function. Most commonly affected are lysosomal …
several components of lysosomal function. Most commonly affected are lysosomal …
Emptying the stores: lysosomal diseases and therapeutic strategies
FM Platt - Nature reviews Drug discovery, 2018 - nature.com
Lysosomal storage disorders (LSDs)—designated as' orphan'diseases—are inborn errors of
metabolism caused by defects in genes that encode proteins involved in various aspects of …
metabolism caused by defects in genes that encode proteins involved in various aspects of …
TFEB at a glance
The transcription factor EB (TFEB) plays a pivotal role in the regulation of basic cellular
processes, such as lysosomal biogenesis and autophagy. The subcellular localization and …
processes, such as lysosomal biogenesis and autophagy. The subcellular localization and …