Silicon Valley Bussiness Journal, Feb 1, 2016, Cromwell Schubarth, TechFlash Editor Silicon Valley Business Journal
Avalanche Biotechnologies has agreed to acquire Paris-based Annapurna Therapeutics in a $105.6 million deal that expands its gene therapy pipeline. The Menlo Park company led by Paul Cleveland said the deal will combine his company's four gene therapy programs for ophthalmic diseases with Annapurna’s gene therapies for Alpha1-antitrypsin (A1AT) deficiency, cardiomyopathy associated with Friedreich’s ataxia, hereditary angioedema, and severe allergies.
Related news (January 5,2016): Annapurna Therapeutics (formerly AAVLife SAS) to Collaborate with Weill Cornell Medicine on Gene-Therapy Portfolio
Tuesday, February 2, 2016
Monday, February 1, 2016
Cth2 Protein Mediates Early Adaptation of Yeast Cells to Oxidative Stress Conditions
Castells-Roca L, Pijuan J, Ferrezuelo F, Bellí G, Herrero E (2016). PLoS ONE 11(1): e0148204. doi:10.1371/journal.pone.0148204
Open Access
Cellular responses to environmental stresses involve changes in RNA transcript levels, through the modulation of the kinetics of transcription and decay rates of individual mRNA molecules.
Open Access
Cellular responses to environmental stresses involve changes in RNA transcript levels, through the modulation of the kinetics of transcription and decay rates of individual mRNA molecules.
Sunday, January 31, 2016
PATENT: METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
Bibliographic data: US2016024526 (A1) ― 2016-01-28
Inventor(s): PUCCIO HELENE MONIQUE [FR]; AUBORG PATRICK [FR]; CRYSTAL RONALD G [US]; BOUGNERES PIERRE [FR]
Applicant(s): APHP ASSISTANCE PUBLIQUE HÔPITAUX DE PARIS [FR]; UNIV CORNELL [US]; INST NAT SANTE RECH MED [FR]; CENTRE NAT RECH SCIENT [FR]; UNIV STRASBOURG [FR]; UNIVERSITÉ PARIS SUD XI [FR]
The invention relates to a method for preventing or treating a cardiomyopathy associated with Friedreich ataxia in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a frataxin (FXN) encoding nucleic acid.
Inventor(s): PUCCIO HELENE MONIQUE [FR]; AUBORG PATRICK [FR]; CRYSTAL RONALD G [US]; BOUGNERES PIERRE [FR]
Applicant(s): APHP ASSISTANCE PUBLIQUE HÔPITAUX DE PARIS [FR]; UNIV CORNELL [US]; INST NAT SANTE RECH MED [FR]; CENTRE NAT RECH SCIENT [FR]; UNIV STRASBOURG [FR]; UNIVERSITÉ PARIS SUD XI [FR]
The invention relates to a method for preventing or treating a cardiomyopathy associated with Friedreich ataxia in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a frataxin (FXN) encoding nucleic acid.
Saturday, January 30, 2016
Role of iron in neurodegenerative diseases
Kai Li, Heinz Reichmann; Neurology and Preclinical Neurological Studies - Review Article, Journal of Neural Transmission pp 1-11 First online: 21 January 2016 DOI: 10.1007/s00702-016-1508-7
This review summarize recent developments on iron dyshomeostasis in Parkinson’s disease, Alzheimer’s disease, Friedreich ataxia, and Huntington’s disease.
This review summarize recent developments on iron dyshomeostasis in Parkinson’s disease, Alzheimer’s disease, Friedreich ataxia, and Huntington’s disease.
Friday, January 29, 2016
The diagnostic value of saccades in movement disorder patients: a practical guide and review
Pichet Termsarasab, Thananan Thammongkolchai, Janet C. Rucker and Steven J. Frucht; Journal of Clinical Movement Disorders 2015 2:14 DOI: 10.1186/s40734-015-0025-4
OPEN ACCESS
Saccades may be very useful diagnostically in recessive forms of cerebellar ataxia. In Friedreich’s ataxia, prominent fixation instability may take the form of macrosaccadic oscillations or nearly continuous square wave jerks, while interestingly cerebellar atrophy is not seen until the very late stages of the illness.
OPEN ACCESS
Saccades may be very useful diagnostically in recessive forms of cerebellar ataxia. In Friedreich’s ataxia, prominent fixation instability may take the form of macrosaccadic oscillations or nearly continuous square wave jerks, while interestingly cerebellar atrophy is not seen until the very late stages of the illness.
Thursday, January 28, 2016
Nrf2 activation in the treatment of neurodegenerative diseases: a focus on its role in mitochondrial bioenergetics and function
Noemí Esteras, Albena T. Dinkova-Kostova, Andrey Y. Abramov; Biological Chemistry. ISSN (Online) 1437-4315, ISSN (Print) 1431-6730, DOI: 10.1515/hsz-2015-0295, January 2016
Currently, REATA Pharmaceuticals has initiated a clinical trial with a novel Nrf2 activator, named RTA 408, for the treatment of Friedreich’s ataxia (ClinicalTrials.gov, NCT02255435). This neurodegenerative disease is caused by deficiency of the protein frataxin, which causes the disruption of iron-sulphur cluster biosynthesis, mitochondrial iron overload and an increased sensitivity to oxidative stress, mainly due to a decrease in the expression of Nrf2.
Currently, REATA Pharmaceuticals has initiated a clinical trial with a novel Nrf2 activator, named RTA 408, for the treatment of Friedreich’s ataxia (ClinicalTrials.gov, NCT02255435). This neurodegenerative disease is caused by deficiency of the protein frataxin, which causes the disruption of iron-sulphur cluster biosynthesis, mitochondrial iron overload and an increased sensitivity to oxidative stress, mainly due to a decrease in the expression of Nrf2.
Wednesday, January 27, 2016
Synthesis, Delivery and Regulation of Eukaryotic Heme and Fe-S Cluster Cofactors
Dulmini P. Barupala, Stephen P. Dzul, Pamela Jo Riggs-Gelasco, Timothy L. Stemmler, Archives of Biochemistry and Biophysics, Available online 16 January 2016, ISSN 0003-9861, doi:10.1016/j.abb.2016.01.010.
With an incidence of 1 in 50,000172,173, and a carrier prevalence of 1 in 100174, Friedreich’s ataxia (FRDA) is by far the most prevalent disease linked to defective Fe-S cluster formation.
FRDA is an autosomal recessive genetic disease caused by a GAA-trinucleotide repeat expansion in an intron of the frataxin gene, a protein involved in the ISC pathway.
With an incidence of 1 in 50,000172,173, and a carrier prevalence of 1 in 100174, Friedreich’s ataxia (FRDA) is by far the most prevalent disease linked to defective Fe-S cluster formation.
FRDA is an autosomal recessive genetic disease caused by a GAA-trinucleotide repeat expansion in an intron of the frataxin gene, a protein involved in the ISC pathway.
Tuesday, January 26, 2016
Measurement Characteristics and Clinical Utility of the International Cooperative Ataxia Rating Scale in Individuals With Hereditary Ataxias
Maryleen K. Jones, Stephanie A. Combs-Miller, Archives of Physical Medicine and Rehabilitation, Volume 97, Issue 2, February 2016, Pages 341-342, ISSN 0003-9993, doi:10.1016/j.apmr.2015.04.002.
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