Therapeutic strategies for the treatment of spinal muscular atrophy.

Abstract:

:Spinal muscular atrophy (SMA) is an inherited neurodegenerative disease that results in progressive dysfunction of motor neurons of the anterior horn of the spinal cord. SMA is caused by the loss of full-length protein expression from the survival of motor neuron 1 (SMN1) gene. The disease has a unique genetic profile as it is autosomal recessive for the loss of SMN1, but a nearly identical homolog, SMN2, acts as a disease modifier whose expression is inversely correlated to clinical severity. Targeted therapeutic approaches primarily focus on increasing the levels of full-length SMN protein, through either gene replacement or regulation of SMN2 expression. There is currently no US FDA approved treatment for SMA. This is an exciting time as multiple efforts from academic and industrial laboratories are reaching the preclinical and clinical testing stages.

journal_name

Future Med Chem

authors

Cherry JJ,Androphy EJ

doi

10.4155/fmc.12.107

subject

Has Abstract

pub_date

2012-09-01 00:00:00

pages

1733-50

issue

13

eissn

1756-8919

issn

1756-8927

journal_volume

4

pub_type

杂志文章,评审
  • Recent advance of the hepatitis B virus inhibitors: a medicinal chemistry overview.

    abstract::Hepatitis B Virus (HBV) is one of the most prevalent viral infections of human worldwide. The therapies are limited in the clinical context because of negative side effects of interferons and the development of viral resistance to the nucleoside/nucleotide inhibitors. In this review, we summarize the recent advances i...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc.15.19

    authors: Jia H,Rai D,Zhan P,Chen X,Jiang X,Liu X

    更新日期:2015-01-01 00:00:00

  • Current and future perspectives on the chemotherapy of the parasitic protozoa Trichomonas vaginalis and Entamoeba histolytica.

    abstract::Trichomonas vaginalis and Entamoeba histolytica are clinically important protozoa that affect humans. T. vaginalis produces sexually transmitted infections and E. histolytica is the causative agent of amebic dysentery. Metronidazole, a compound first used to treat T. vaginalis in 1959, is still the main drug used worl...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc.09.59

    authors: Moya IA,Su Z,Honek JF

    更新日期:2009-07-01 00:00:00

  • Cyclic endomorphin analogs in targeting opioid receptors to achieve pain relief.

    abstract::Endomorphins, the endogenous ligands of the µ-opioid receptor, are attractive candidates for opioid-based pain-relieving agents. These tetrapeptides, with their remarkable affinity for the µ-opioid receptor, display favorable antinociceptive activity when injected directly into the brain of experimental animals. Howev...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc.14.132

    authors: Janecka A,Gentilucci L

    更新日期:2014-01-01 00:00:00

  • Gallium-containing anticancer compounds.

    abstract::There is an ever pressing need to develop new drugs for the treatment of cancer. Gallium nitrate, a group IIIa metal salt, inhibits the proliferation of tumor cells in vitro and in vivo and has shown activity against non-Hodgkin's lymphoma and bladder cancer in clinical trials. Gallium can function as an iron mimetic ...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc.12.69

    authors: Chitambar CR

    更新日期:2012-06-01 00:00:00

  • Recent advances and new perspectives in targeting CFTR for therapy of cystic fibrosis and enterotoxin-induced secretory diarrheas.

    abstract::The cystic fibrosis transmembrane conductance regulator (CFTR) is a cAMP-regulated chloride channel localized primarily at the apical surfaces of epithelial cells lining airway, gut and exocrine glands, where it is responsible for transepithelial salt and water transport. Several human diseases are associated with an ...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc.12.1

    authors: Zhang W,Fujii N,Naren AP

    更新日期:2012-03-01 00:00:00

  • Interview with Solomon Nwaka.

    abstract::Solomon Nwaka was born in Nigeria and grew up seeing at first hand the impact of neglected diseases. His research efforts have taken him across the globe, in both the developed and developing world. Following posts in academia, industry and at the Medicines for Malaria Venture, he is now at the WHO where he is engaged...

    journal_title:Future medicinal chemistry

    pub_type: 面试

    doi:10.4155/fmc.11.99

    authors: Nwaka S

    更新日期:2011-09-01 00:00:00

  • A survey of the mechanisms of action of anticancer transition metal complexes.

    abstract::Metal complexes have been the subject of numerous investigations in oncology but, despite the plethora of newly synthesized compounds, their precise mechanisms of action remain generally unknown or, for the best, incompletely determined. The continuous development of efficient and sensitive techniques in analytical ch...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc-2016-0153

    authors: Marloye M,Berger G,Gelbcke M,Dufrasne F

    更新日期:2016-12-01 00:00:00

  • New preparation and synthetic reactions of 3,3,3-trifluoropropynyllithium, -borate and -stannane: facile synthesis of trifluoromethylated allenes, arylacetylenes and enynes.

    abstract:BACKGROUND:Since trifluoromethyl-containing compounds have found diverse applications in the fields of pharmaceuticals and agrochemicals, facile and selective synthetic methods for trifluoromethyl-substituted compounds are an essential tool for advanced medicinal chemistry. Now that diverse synthetic transformations of...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章

    doi:10.4155/fmc.09.69

    authors: Shimizu M,Higashi M,Takeda Y,Murai M,Jiang G,Asai Y,Nakao Y,Shirakawa E,Hiyama T

    更新日期:2009-08-01 00:00:00

  • DYRK1A inhibition as potential treatment for Alzheimer's disease.

    abstract::In total, 47,500,000 people worldwide are affected by dementia and this number is estimated to double by 2030 and triple within 2050 resulting in a huge burden on public health. Alzheimer's disease (AD), a progressive neurodegenerative disorder, is the most common cause of dementia, accounting for 60-70% of all the ca...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc-2016-0013

    authors: Stotani S,Giordanetto F,Medda F

    更新日期:2016-04-01 00:00:00

  • Structural and functional regulation of lactate dehydrogenase-A in cancer.

    abstract::Dysregulated metabolism is one of the hallmarks of cancer. Under normal physiological conditions, ATP is primarily generated by oxidative phosphorylation. Cancers commonly undergo a dramatic shift toward glycolysis, despite the presence of oxygen. This phenomenon is known as the Warburg effect, and requires the activi...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章

    doi:10.4155/fmc-2019-0287

    authors: Woodford MR,Chen VZ,Backe SJ,Bratslavsky G,Mollapour M

    更新日期:2020-03-01 00:00:00

  • Rho kinase as a target for cerebral vascular disorders.

    abstract::The development of novel pharmaceutical treatments for disorders of the cerebral vasculature is a serious unmet medical need. These vascular disorders are typified by a disruption in the delicate Rho signaling equilibrium within the blood vessel wall. In particular, Rho kinase overactivation in the smooth muscle and e...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc.15.45

    authors: Bond LM,Sellers JR,McKerracher L

    更新日期:2015-01-01 00:00:00

  • Synthesis of ring-substituted N-benzyl γ-phenyl GABA derivatives via reductive amination.

    abstract:BACKGROUND:GABA derivatives are important for the development of drugs for disorders of the CNS and PNS. RESULTS:We describe a simple synthesis of N-benzyl γ-phenyl-γ-aminobutyric acids, via the reductive amination of the corresponding γ-keto acids with benzylamines using ammonia borane. CONCLUSION:This procedure pro...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章

    doi:10.4155/fmc.10.300

    authors: Ramachandran PV,Sakavuyi K,Mistry H

    更新日期:2011-02-01 00:00:00

  • Antitumoral activity of 1,2-diaminocyclohexane derivatives in breast, colon and skin human cancer cells.

    abstract:AIM:Cancer is among the leading causes of death worldwide. Medical interest has focused on macrocyclic polyamines because of their properties as antitumor agents. Results/Methodology: We have designed and synthesized a series of 1,2-diaminocyclohexane derivatives with notable in vitro antiproliferative activities again...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章

    doi:10.4155/fmc-2016-0212

    authors: Morales F,Ramírez A,Morata-Tarifa C,Navarro SA,Marchal JA,Campos JM,Conejo-García A

    更新日期:2017-03-01 00:00:00

  • Selective and potent small-molecule inhibitors of PI3Ks.

    abstract::Class I PI3Ks are composed of four catalytic subunit variants (p110α, p110β, p110δ and p110γ). The PI3K pathway is among the most frequently activated pathways in many diseases, and has emerged as an attractive target for drug development, in particular for the treatment of many human cancers including breast, prostat...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc.14.28

    authors: Jeong Y,Kwon D,Hong S

    更新日期:2014-05-01 00:00:00

  • N3-substituted thymidine bioconjugates for cancer therapy and imaging.

    abstract::The compound class of 3-carboranyl thymidine analogues (3CTAs) are boron delivery agents for boron neutron capture therapy (BNCT), a binary treatment modality for cancer. Presumably, these compounds accumulate selectively in tumor cells via intracellular trapping, which is mediated by hTK1. Favorable in vivo biodistri...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc.13.31

    authors: Khalil A,Ishita K,Ali T,Tjarks W

    更新日期:2013-04-01 00:00:00

  • Microvascular complications and diabetic retinopathy: recent advances and future implications.

    abstract::Retinal microvascular alterations have been observed during diabetic retinopathy (DR) due to the retinal susceptibility towards subtle pathological alterations. Therefore, retinal microvascular pathology is essential to understand the nature of retinal degenerations during DR. In this review, the role of retinal micro...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc.12.206

    authors: Barot M,Gokulgandhi MR,Patel S,Mitra AK

    更新日期:2013-03-01 00:00:00

  • Identification of agents targeting FtsZ assembly.

    abstract::Filamenting temperature-sensitive mutant Z (FtsZ), an essential cell division protein in bacteria, has recently emerged as an important and exploitable antibacterial target. Cytokinesis in bacteria is regulated by the assembly dynamics of this protein, which is ubiquitously present in eubacteria. The perturbation of F...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc-2016-0041

    authors: Panda D,Bhattacharya D,Gao QH,Oza PM,Lin HY,Hawkins B,Hibbs DE,Groundwater PW

    更新日期:2016-06-01 00:00:00

  • Multitarget strategies in Alzheimer's disease: benefits and challenges on the road to therapeutics.

    abstract::Alzheimer's disease is a multifactorial syndrome, for which effective cures are urgently needed. Seeking for enhanced therapeutic efficacy, multitarget drugs have been increasingly sought after over the last decades. They offer the attractive prospect of tackling intricate network effects, but with the benefits of a s...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc-2016-0003

    authors: Rosini M,Simoni E,Caporaso R,Minarini A

    更新日期:2016-04-01 00:00:00

  • Hydroxylation of protein constituents of the human translation system: structural aspects and functional assignments.

    abstract::During the current decade, data on the post-translational hydroxylation of specific amino acid residues of some ribosomal proteins and translation factors in both eukaryotes and eubacteria have accumulated. The reaction is catalyzed by dedicated oxygenases (so-called ribosomal oxygenases), whose action is impaired und...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章

    doi:10.4155/fmc-2018-0317

    authors: Graifer D,Malygin A,Karpova G

    更新日期:2019-02-25 00:00:00

  • Estimation of kinetic and thermodynamic ligand-binding parameters using computational strategies.

    abstract::Kinetic and thermodynamic ligand-protein binding parameters are gaining growing importance as key information to consider in drug discovery. The determination of the molecular structures, using particularly x-ray and NMR techniques, is crucial for understanding how a ligand recognizes its target in the final binding c...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc-2016-0224

    authors: Deganutti G,Moro S

    更新日期:2017-04-01 00:00:00

  • A beginner's guide to chemokines.

    abstract::This review provides an overview of chemokines and their receptors, with an emphasis on general features and nomenclature along with a short summary of their properties and functions. It is intended as an introduction to the subject and a reference point for those wishing to learn key facts about chemokines and their ...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc.12.49

    authors: Vinader V,Afarinkia K

    更新日期:2012-05-01 00:00:00

  • Discovery of anti-TB agents that target the cell-division protein FtsZ.

    abstract::The emergence of multidrug-resistant Mycobacterium tuberculosis strains has made many of the currently available anti-tuberculosis (TB) drugs ineffective. Accordingly, there is a pressing need to identify new drug targets. Filamentous temperature-sensitive protein Z (FtsZ), a bacterial tubulin homologue, is an essenti...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc.10.220

    authors: Kumar K,Awasthi D,Berger WT,Tonge PJ,Slayden RA,Ojima I

    更新日期:2010-08-01 00:00:00

  • Pharmacokinetic strategies for treatment of drug overdose and addiction.

    abstract::The pharmacokinetic treatment strategy targets the drug molecule itself, aiming to reduce drug concentration at the site of action, thereby minimizing any pharmacodynamic effect. This approach might be useful in the treatment of acute drug toxicity/overdose and in the long-term treatment of addiction. Phase IIa contro...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc.11.190

    authors: Gorelick DA

    更新日期:2012-02-01 00:00:00

  • Small molecules as friends and foes of the immune system.

    abstract::Every organism is in contact with numerous small molecules (<1000 Da). Chemicals may cause or trigger adverse health effects, including diseases of the immune system. They may also be exploited as drugs. In this review, we look at the interaction between small molecules and the immune system. We discuss the hapten and...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc.09.125

    authors: Kadow S,Jux B,Chmill S,Esser C

    更新日期:2009-12-01 00:00:00

  • Strategies for the treatment of dengue virus infections: a narrative account.

    abstract::In comparison with other virus infections, such as HIV, HBV, HCV, influenza and herpes virus, dengue virus infections have received relatively little attention from a (chemo)therapeutic viewpoint, despite their global incidence (2.5 billion people estimated to be at risk) and absence of an effective prophylactic measu...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章

    doi:10.4155/fmc.10.15

    authors: De Clercq E

    更新日期:2010-04-01 00:00:00

  • In silico and saturation transfer difference NMR approaches to unravel the binding mode of an andrographolide derivative to K-Ras oncoprotein.

    abstract::Background: Andrographolide and its benzylidene derivatives, SRJ09 and SRJ23, potentially bind oncogenic K-Ras to exert anticancer activity. Their molecular interactions with K-Ras oncoproteins that lead to effective biological activity are of major interest. Methods & results:In silico docking and molecular dynamics ...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章

    doi:10.4155/fmc-2020-0104

    authors: Quah SY,Tan MS,Ho KL,Manan NA,Gorfe AA,Deb PK,Sagineedu SR,Stanslas J

    更新日期:2020-09-01 00:00:00

  • Recent advances in the discovery of small molecule mTOR inhibitors.

    abstract::Mammalian target of rapamycin (mTOR) belongs to the atypical kinase family of phosphatidylinositol-3-kinase-related kinase and function as a master regulators of the switch between catabolic and anabolic metabolism. In the last decade mTOR has emerged as a therapeutic target for various diseases such as cancer, inflam...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc.10.233

    authors: Roychowdhury A,Sharma R,Kumar S

    更新日期:2010-10-01 00:00:00

  • Selection and optimization of hits from a high-throughput phenotypic screen against Trypanosoma cruzi.

    abstract:BACKGROUND:Inhibitors of Trypanosoma cruzi with novel mechanisms of action are urgently required to diversify the current clinical and preclinical pipelines. Increasing the number and diversity of hits available for assessment at the beginning of the discovery process will help to achieve this aim. RESULTS:We report t...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章

    doi:10.4155/fmc.13.139

    authors: Keenan M,Alexander PW,Chaplin JH,Abbott MJ,Diao H,Wang Z,Best WM,Perez CJ,Cornwall SM,Keatley SK,Thompson RC,Charman SA,White KL,Ryan E,Chen G,Ioset JR,von Geldern TW,Chatelain E

    更新日期:2013-10-01 00:00:00

  • The dextrans as vehicles for gene and drug delivery.

    abstract::Dextran has become a hot research topic in drug vehicle material because of its biodegradable, nonspecific cell adhesion, resistance to protein adsorption, low price and ease of structural modification. The fate and changes of dextran in vivo are not fully understood. It is helpful to guide the design and modification...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc-2018-0586

    authors: Huang S,Huang G

    更新日期:2019-07-01 00:00:00

  • Anti-inflammatory and antioxidative effects of Camellia oleifera Abel components.

    abstract::Camellia oleifera Abel is a member of Camellia, and its seeds are used to extract Camellia oil, which is generally used as cooking oil in the south of China. Camellia oil consists of unsaturated fatty acids, tea polyphenol, squalene, saponin, carrot element and vitamins, etc. The seed remains after oil extraction of C...

    journal_title:Future medicinal chemistry

    pub_type: 杂志文章,评审

    doi:10.4155/fmc-2017-0109

    authors: Xiao X,He L,Chen Y,Wu L,Wang L,Liu Z

    更新日期:2017-11-01 00:00:00