Natural products: an evolving role in future drug discovery
The therapeutic areas of infectious diseases and oncology have benefited from abundant
scaffold diversity in natural products, able to interact with many specific targets within the cell …
scaffold diversity in natural products, able to interact with many specific targets within the cell …
Natural products to drugs: natural product-derived compounds in clinical trials
MS Butler - Natural product reports, 2008 - pubs.rsc.org
Covering: 2005 to 2007 Natural product and natural product-derived compounds that are
being evaluated in clinical trials or are in registration (as at 31st December 2007) have been …
being evaluated in clinical trials or are in registration (as at 31st December 2007) have been …
Pompe's disease
AT van der Ploeg, AJJ Reuser - The lancet, 2008 - thelancet.com
Pompe's disease, glycogen-storage disease type II, and acid maltase deficiency are
alternative names for the same metabolic disorder. It is a pan-ethnic autosomal recessive …
alternative names for the same metabolic disorder. It is a pan-ethnic autosomal recessive …
Transcription factor EB (TFEB) is a new therapeutic target for Pompe disease
C Spampanato, E Feeney, L Li, M Cardone… - EMBO molecular …, 2013 - embopress.org
A recently proposed therapeutic approach for lysosomal storage disorders (LSDs) relies
upon the ability of transcription factor EB (TFEB) to stimulate autophagy and induce …
upon the ability of transcription factor EB (TFEB) to stimulate autophagy and induce …
Pharmacological chaperone therapy: preclinical development, clinical translation, and prospects for the treatment of lysosomal storage disorders
Lysosomal storage disorders (LSDs) are a group of inborn metabolic diseases caused by
mutations in genes that encode proteins involved in different lysosomal functions, in most …
mutations in genes that encode proteins involved in different lysosomal functions, in most …
Iminosugars past, present and future: medicines for tomorrow
G Horne, FX Wilson, J Tinsley, DH Williams… - Drug discovery today, 2011 - Elsevier
Iminosugars comprise the most attractive class of carbohydrate mimetics reported to date
and are ideally positioned to take advantage of our increasing understanding of …
and are ideally positioned to take advantage of our increasing understanding of …
Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics
G Parenti - EMBO molecular medicine, 2009 - embopress.org
Lysosomal storage diseases (LSDs) are a group of genetic disorders due to defects in any
aspect of lysosomal biology. During the past two decades, different approaches have been …
aspect of lysosomal biology. During the past two decades, different approaches have been …
Pharmacological chaperones as therapeutics for lysosomal storage diseases
RE Boyd, G Lee, P Rybczynski… - Journal of medicinal …, 2013 - ACS Publications
Lysosomal enzymes are responsible for the degradation of a wide variety of glycolipids,
oligosaccharides, proteins, and glycoproteins. Inherited mutations in the genes that encode …
oligosaccharides, proteins, and glycoproteins. Inherited mutations in the genes that encode …
Identification and characterization of pharmacological chaperones to correct enzyme deficiencies in lysosomal storage disorders
KJ Valenzano, R Khanna, AC Powe Jr… - Assay and drug …, 2011 - liebertpub.com
Many human diseases result from mutations in specific genes. Once translated, the resulting
aberrant proteins may be functionally competent and produced at near-normal levels …
aberrant proteins may be functionally competent and produced at near-normal levels …
Chaperoning G protein-coupled receptors: from cell biology to therapeutics
YX Tao, PM Conn - Endocrine reviews, 2014 - academic.oup.com
G protein-coupled receptors (GPCRs) are membrane proteins that traverse the plasma
membrane seven times (hence, are also called 7TM receptors). The polytopic structure of …
membrane seven times (hence, are also called 7TM receptors). The polytopic structure of …