Distillery: Therapeutics
June 25 2009, Volume 2 / Number 25
Autoimmune disease
Rheumatoid arthritis (RA)
Proteasome prosome macropain subunit-
type 8 (PSMB8; LMP7)
doi:10.1038/scibx.2009.997
Cell culture and mouse studies suggest that inhibiting LMP7 could be useful for treating RA.
Summary - Proteasome prosome macropain subunit-
type 8 (PSMB8; LMP7) | PDF (114 KB) - Proteasome prosome macropain subunit- type 8 (PSMB8; LMP7)
Cancer
Acute lymphoblastic leukemia (ALL)
CC chemokine receptor 7 (CCR7); chemokine C-C motif ligand 19 (CCL19); Notch homolog 1 translocation-associated (NOTCH1)
doi:10.1038/scibx.2009.998
Studies in mice suggest that antagonizing CCR7 could help prevent the spread of ALL to the CNS.
Summary - CC chemokine receptor 7 (CCR7); chemokine C-C motif ligand 19 (CCL19); Notch homolog 1 translocation-associated (NOTCH1) | PDF (110 KB) - CC chemokine receptor 7 (CCR7); chemokine C-C motif ligand 19 (CCL19); Notch homolog 1 translocation-associated (NOTCH1)
Cancer
Cyclin-dependent kinase 2 (CDK2); v-myc myelocytomatosis viral related oncogene, neuroblastoma derived (MYCN)
doi:10.1038/scibx.2009.999
A study in cell culture suggests that inhibiting CDK2 could help treat MYCN-overexpressing cancers.
Summary - Cyclin-dependent kinase 2 (CDK2); v-myc myelocytomatosis viral related oncogene, neuroblastoma derived (MYCN) | PDF (113 KB) - Cyclin-dependent kinase 2 (CDK2); v-myc myelocytomatosis viral related oncogene, neuroblastoma derived (MYCN)
Cancer
Integrin 
v3
doi:10.1038/scibx.2009.1000
A study in mice suggests that inhibiting integrin 
v3 could be useful for preventing cancer metastasis to the brain.
Cancer
Not applicable
doi:10.1038/scibx.2009.1001
In vitro studies suggest that carbohydrate conjugates of the photosensitizing agent 2-(1'-hexyloxyethyl)-2-devinylpyropheophorbide-a (HPPH) may be useful phototherapies for cancer.
Cancer
Unknown
doi:10.1038/scibx.2009.1002
Studies in cell culture identified a [3]ferrocenophane polyphenol that could be useful for treating cancer.
Colorectal cancer
NDRG family member 4 (NDRG4)
doi:10.1038/scibx.2009.1003
Studies in human tissues and in cell lines suggest that the tumor suppressor NDRG4 could be a marker for colorectal cancer.
Summary - NDRG family member 4 (NDRG4) | PDF (114 KB) - NDRG family member 4 (NDRG4)
Epithelial cancer
Cytoplasmic FMR1-interacting protein 1 (CYFIP1)
doi:10.1038/scibx.2009.1004
Patient samples and studies in mice suggest that CYFIP1 functions as a tumor suppressor and that enhancing its activity could help treat epithelial cancers.
Summary - Cytoplasmic FMR1-interacting protein 1 (CYFIP1) | PDF (110 KB) - Cytoplasmic FMR1-interacting protein 1 (CYFIP1)
Cardiovascular disease
Cardiac hypertrophy
Hypoxia-inducible factor 1 (HIF1A; HIF1); peroxisome proliferation–activated receptor-
(PPARG; PPAR)
doi:10.1038/scibx.2009.1005
Studies in mice and in cell culture suggest that inhibiting HIF1 could be useful for preventing cardiac hypertrophy.
Summary - Hypoxia-inducible factor 1 (HIF1A; HIF1); peroxisome proliferation–activated receptor-
(PPARG; PPAR) | PDF (113 KB) - Hypoxia-inducible factor 1 (HIF1A; HIF1); peroxisome proliferation–activated receptor- (PPARG; PPAR)
Fibrillation
Calcium calmodulin-dependent protein kinase II (CAMK2); ryanodine receptor 2 (RyR2)
doi:10.1038/scibx.2009.1006
In vivo and tissue studies suggest that antagonizing CAMK2 could help treat atrial fibrillation.
Summary - Calcium calmodulin-dependent protein kinase II (CAMK2); ryanodine receptor 2 (RyR2) | PDF (112 KB) - Calcium calmodulin-dependent protein kinase II (CAMK2); ryanodine receptor 2 (RyR2)
Heart failure
Unknown
doi:10.1038/scibx.2009.1007
Studies in canines and in tissue culture suggest that a modified B-type natriuretic peptide (ASBNP.1) could help treat heart failure with fewer renal side effects than first-generation peptides.
Hypercholesterolemia
Liver X receptor- (NR1H3; LXR-); NR1H2 (LXR-); myosin regulatory light chain interacting protein (MYLIP; IDOL)
doi:10.1038/scibx.2009.1008
A study in human and mouse cell culture suggests that antagonizing IDOL could help lower cholesterol.
Summary - Liver X receptor- (NR1H3; LXR-); NR1H2 (LXR-); myosin regulatory light chain interacting protein (MYLIP; IDOL) | PDF (114 KB) - Liver X receptor- (NR1H3; LXR-); NR1H2 (LXR-); myosin regulatory light chain interacting protein (MYLIP; IDOL)
Endocrine disease
Diabetes
AMP-activated protein kinase (AMPK)
doi:10.1038/scibx.2009.1009
A sulfonamide prodrug of generic metformin could be useful for reducing the daily dosage of the drug and decreasing gastrointestinal side effects in type 2 diabetes patients.
Summary - AMP-activated protein kinase (AMPK) | PDF (111 KB) - AMP-activated protein kinase (AMPK)
Diabetes
Sirtuin 1 (SIRT1)
doi:10.1038/scibx.2009.1010
Studies in rats suggest that inhibiting hepatic SIRT1 could help treat type 2 diabetes.
Summary - Sirtuin 1 (SIRT1) | PDF (112 KB) - Sirtuin 1 (SIRT1)
Diabetes; obesity
Leptin receptor (LEPR)
doi:10.1038/scibx.2009.1011
Studies in mice suggest that boosting LEPR activity on proopiomelanocortin (POMC) neurons could help prevent diabetes and obesity.
Summary - Leptin receptor (LEPR) | PDF (111 KB) - Leptin receptor (LEPR)
Infectious disease
Enterovirus
P selectin glycoprotein ligand-1 (SELPLG; PSGL-1; CD162); scavenger receptor class B member 2 (SCARB2)
doi:10.1038/scibx.2009.1012
Two cell-culture studies suggest that targeting PSGL-1 and SCARB2 could help treat hand, foot and mouth disease caused by enterovirus 71 (EV71).
Summary - P selectin glycoprotein ligand-1 (SELPLG; PSGL-1; CD162); scavenger receptor class B member 2 (SCARB2) | PDF (113 KB) - P selectin glycoprotein ligand-1 (SELPLG; PSGL-1; CD162); scavenger receptor class B member 2 (SCARB2)
Infectious disease
Mammalian target of rapamycin (mTOR; FRAP; RAFT1)
doi:10.1038/scibx.2009.1013
Studies in mice and in macaques suggest that rapamycin could help increase the efficacy of infectious disease vaccines.
Summary - Mammalian target of rapamycin (mTOR; FRAP; RAFT1) | PDF (112 KB) - Mammalian target of rapamycin (mTOR; FRAP; RAFT1)
Influenza virus
Influenza A virus neuraminidase
doi:10.1038/scibx.2009.1014
Studies in cell culture suggest that porphyrin-zanamivir conjugates are photodynamic therapeutics for influenza A infection.
Summary - Influenza A virus neuraminidase | PDF (112 KB) - Influenza A virus neuraminidase
Malaria
Plasmodium falciparum heat shock protein 101 (Hsp101); P. falciparum hypothetical protein (PF14_0344; Ptex150); P. falciparum exported protein 2 (PKG_123420; Exp2); P. falciparum hypothetical protein (PF11_0067; Ptex88); P. falciparum mitochondrial thioredoxin (MAL13P1.225; Trx2)
doi:10.1038/scibx.2009.1015
In vitro studies identified a P. falciparum protein trafficking complex that could be targeted to help treat malaria.
Summary - Plasmodium falciparum heat shock protein 101 (Hsp101); P. falciparum hypothetical protein (PF14_0344; Ptex150); P. falciparum exported protein 2 (PKG_123420; Exp2); P. falciparum hypothetical protein (PF11_0067; Ptex88); P. falciparum mitochondrial thioredoxin (MAL13P1.225; Trx2) | PDF (112 KB) - Plasmodium falciparum heat shock protein 101 (Hsp101); P. falciparum hypothetical protein (PF14_0344; Ptex150); P. falciparum exported protein 2 (PKG_123420; Exp2); P. falciparum hypothetical protein (PF11_0067; Ptex88); P. falciparum mitochondrial thioredoxin (MAL13P1.225; Trx2)
Viral infection
Zinc finger and BTB domain containing 16 (ZBTB16; PLZF)
doi:10.1038/scibx.2009.1016
Studies in mice suggest that enhancing PLZF expression could help treat viral infection.
Summary - Zinc finger and BTB domain containing 16 (ZBTB16; PLZF) | PDF (112 KB) - Zinc finger and BTB domain containing 16 (ZBTB16; PLZF)
Metabolic disease
Metabolic disorders
ATP-binding cassette sub-family G WHITE member 2 (ABCG2; MXR)
doi:10.1038/scibx.2009.1017
Cell culture and population studies suggest that enhancing ABCG2 activity could help lower urate levels and treat gout.
Summary - ATP-binding cassette sub-family G WHITE member 2 (ABCG2; MXR) | PDF (114 KB) - ATP-binding cassette sub-family G WHITE member 2 (ABCG2; MXR)
Musculoskeletal disease
Muscular atrophy
Survival of motor neuron 1 telomeric (SMN1; SMN); survival of motor neuron 2 centromeric (SMN2; SMNC)
doi:10.1038/scibx.2009.1018
Studies in mice suggest that an antisense oligonucleotide targeting the SMN2 gene could increase levels of SMN and help treat spinal muscular atrophy.
Summary - Survival of motor neuron 1 telomeric (SMN1; SMN); survival of motor neuron 2 centromeric (SMN2; SMNC) | PDF (113 KB) - Survival of motor neuron 1 telomeric (SMN1; SMN); survival of motor neuron 2 centromeric (SMN2; SMNC)
Neurology
Huntington's disease (HD)
Huntingtin (HTT; HD); Rhes (RASD2)
doi:10.1038/scibx.2009.1019
Cell-culture studies suggest that blocking the interaction between Rhes and mutant HTT could help treat HD.
Summary - Huntingtin (HTT; HD); Rhes (RASD2) | PDF (112 KB) - Huntingtin (HTT; HD); Rhes (RASD2)
Traumatic brain injury
Adenosine A2A receptor (ADORA2A)
doi:10.1038/scibx.2009.1020
A study in murine and human cell cultures suggests that antagonizing ADORA2A could help stimulate microglial activation to treat brain injury.
Summary - Adenosine A2A receptor (ADORA2A) | PDF (116 KB) - Adenosine A2A receptor (ADORA2A)
Renal disease
Diabetic neuropathy
MicroRNA-216a (miRNA-216a); miRNA-217
doi:10.1038/scibx.2009.1021
In vitro studies suggest that inhibiting miRNA-216a or miRNA-217 could help treat kidney disorders.
Summary - MicroRNA-216a (miRNA-216a); miRNA-217 | PDF (110 KB) - MicroRNA-216a (miRNA-216a); miRNA-217
Various
Bone marrow transplant; leukemia
Peroxisome proliferation–activated receptor- (PPARG; PPAR)
doi:10.1038/scibx.2009.1022
A study in mice suggests that antagonizing PPAR could enhance bone marrow transplant engraftment.
Summary - Peroxisome proliferation–activated receptor- (PPARG; PPAR) | PDF (110 KB) - Peroxisome proliferation–activated receptor- (PPARG; PPAR)
