Nature Biotechnology Volume: 29,Pages: 95–97 Year published:(2011) doi:10.1038/nbt0211-95
George S Mack
Maybe not as fast as we would like, but progress is being made in stem cell therapies for neurological diseases.
Tuesday, February 8, 2011
Thursday, February 3, 2011
Gene Therapy Moves Forward in 2010
Molecular Therapy (2011) 19 2, 219–220. doi:10.1038/mt.2010.307
Seppo Ylä-Herttuala
Deputy Editor, and President (2011–2012), European Society of Gene and Cell Therapy
Good summary of recent advances in gene therapy, lists of the most important achievements in this field of knowledge over the past year and shows the growing interest of pharmaceutical companies in hereditary diseases, including many rare diseases.
Seppo Ylä-Herttuala
Deputy Editor, and President (2011–2012), European Society of Gene and Cell Therapy
Good summary of recent advances in gene therapy, lists of the most important achievements in this field of knowledge over the past year and shows the growing interest of pharmaceutical companies in hereditary diseases, including many rare diseases.
Wednesday, February 2, 2011
(WO/2011/009890) USE OF AZABICYCLOALKYL DERIVATIVES OR PYRROLIDINE-2-ONE DERIVATIVES
Patent WO/2011/009890, 27.01.2011,
Aplicants: NOVARTIS AG [CH/CH]; FEUERBACH, Dominik [DE/CH]; GOMEZ-MANCILLA, Baltazar [MX/CH].
The invention concerns the use of azabicydoalkyl derivatives or pyrroiidine-2-one derivatives for the treatment, prevention or delay of progression of ataxia.
"FA, is one of the types of ataxia for which the applicants assume that this drug can be useful"
Aplicants: NOVARTIS AG [CH/CH]; FEUERBACH, Dominik [DE/CH]; GOMEZ-MANCILLA, Baltazar [MX/CH].
The invention concerns the use of azabicydoalkyl derivatives or pyrroiidine-2-one derivatives for the treatment, prevention or delay of progression of ataxia.
"FA, is one of the types of ataxia for which the applicants assume that this drug can be useful"
"Proof of principle", Gene therapy by allele selection in a mouse model of beta-thalassemia
J Clin Invest. 2011;121(2):623–627. doi:10.1172/JCI45377.(February 1, 2011).
Sigrid Eckardt1, N. Adrian Leu2, Ashley Yanchik2, Seigo Hatada3, Michael Kyba4 and K. John McLaughlin1,5
1Center for Molecular and Human Genetics, The Research Institute at Nationwide Children’s Hospital, Columbus, Ohio, USA.
2University of Pennsylvania School of Veterinary Medicine, Philadelphia, Pennsylvania, USA.
3Department of Pathology and Laboratory Medicine, University of North Carolina, Chapel Hill, North Carolina, USA.
4Lillehei Heart Institute and Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.
5Department of Pediatrics, College of Medicine, The Ohio State University, Columbus, Ohio, USA.
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"genetic correction strategy without gene targeting"
"In the case of recessive diseases, MHC-homozygous, disease-free PG ES cells derived from donated oocytes from a heterozygous mother may also generate useful pluripotent cells for treatment in their affected offspring."
Sigrid Eckardt1, N. Adrian Leu2, Ashley Yanchik2, Seigo Hatada3, Michael Kyba4 and K. John McLaughlin1,5
1Center for Molecular and Human Genetics, The Research Institute at Nationwide Children’s Hospital, Columbus, Ohio, USA.
2University of Pennsylvania School of Veterinary Medicine, Philadelphia, Pennsylvania, USA.
3Department of Pathology and Laboratory Medicine, University of North Carolina, Chapel Hill, North Carolina, USA.
4Lillehei Heart Institute and Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.
5Department of Pediatrics, College of Medicine, The Ohio State University, Columbus, Ohio, USA.
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"genetic correction strategy without gene targeting"
"In the case of recessive diseases, MHC-homozygous, disease-free PG ES cells derived from donated oocytes from a heterozygous mother may also generate useful pluripotent cells for treatment in their affected offspring."
Tuesday, February 1, 2011
Johns Hopkins Researchers Develop Safer Way To Make Induced Pluripotent Stem Cells
Stem Cell Research News From Medical News Today, Article Date: 01 Feb 2011 - 4:00 PST
Researchers at Johns Hopkins have found a better way to create induced pluripotent stem (iPS) cells-adult cells reprogrammed with the properties of embryonic stem cells-from a small blood sample. This new method, described last week in Cell Research, avoids creating DNA changes that could lead to tumor formation. read more....
Researchers at Johns Hopkins have found a better way to create induced pluripotent stem (iPS) cells-adult cells reprogrammed with the properties of embryonic stem cells-from a small blood sample. This new method, described last week in Cell Research, avoids creating DNA changes that could lead to tumor formation. read more....
Sunday, January 30, 2011
Medtronic Announces Global Launch Of The CD HORIZON(R) SOLERA™ Spinal System
Medical News Today, Article Date: 30 Jan 2011 - 0:00 PST, Source: Spine Business, Medtronic
Continuing a stream of recent advancements for stabilization of the spine, Medtronic, Inc. (NYSE: MDT) announced both the release of its CD HORIZON® SOLERA™ Spinal System in the U.S. and a limited market release in Japan. This product launch is part of the CD HORIZON® family of fixation devices, designed to provide spinal stabilization and correction as an adjunct to fusion in patients suffering from painful and function-limiting disorders of the middle and lower back. read full text....
Further information:
Continuing a stream of recent advancements for stabilization of the spine, Medtronic, Inc. (NYSE: MDT) announced both the release of its CD HORIZON® SOLERA™ Spinal System in the U.S. and a limited market release in Japan. This product launch is part of the CD HORIZON® family of fixation devices, designed to provide spinal stabilization and correction as an adjunct to fusion in patients suffering from painful and function-limiting disorders of the middle and lower back. read full text....
Further information:
Friday, January 28, 2011
Detection of interruptions in the GAA trinucleotide repeat expansion in the FXN gene of Friedreich ataxia
BioTechniques, Vol. 50, No. 3, March 2011
Timothy P. Holloway*1,2, Simone M. Rowley*1, Martin B. Delatycki1,3, 4, and Joseph P. Sarsero1,2, 3
1Bruce Lefroy Centre for Genetic Health Research, Murdoch Childrens Research Institute, Royal Children's Hospital, Parkville, Victoria, Australia
2Cell and Gene Therapy, Murdoch Childrens Research Institute, Royal Children's Hospital, Parkville, Victoria, Australia
3Department of Paediatrics, The University of Melbourne, Royal Children's Hospital, Parkville, Victoria, Australia
4Department of Clinical Genetics, Austin Health, Heidelberg, Victoria, Australia
"simple and rapid PCR- and restriction enzyme–based assay", "Interruptions in the GAA repeat may serve to alleviate the inhibitory effects of the GAA expansion on FXN gene expression and to decrease pathogenicity"
Full text pdf
Timothy P. Holloway*1,2, Simone M. Rowley*1, Martin B. Delatycki1,3, 4, and Joseph P. Sarsero1,2, 3
1Bruce Lefroy Centre for Genetic Health Research, Murdoch Childrens Research Institute, Royal Children's Hospital, Parkville, Victoria, Australia
2Cell and Gene Therapy, Murdoch Childrens Research Institute, Royal Children's Hospital, Parkville, Victoria, Australia
3Department of Paediatrics, The University of Melbourne, Royal Children's Hospital, Parkville, Victoria, Australia
4Department of Clinical Genetics, Austin Health, Heidelberg, Victoria, Australia
"simple and rapid PCR- and restriction enzyme–based assay", "Interruptions in the GAA repeat may serve to alleviate the inhibitory effects of the GAA expansion on FXN gene expression and to decrease pathogenicity"
Full text pdf
Thursday, January 27, 2011
Mammalian Frataxin: An Essential Function for Cellular Viability through an Interaction with a Preformed ISCU/NFS1/ISD11 Iron-Sulfur Assembly Complex
PLoS ONE 6(1): e16199. doi:10.1371/journal.pone.0016199
Stéphane Schmucker1,2,3,4,5, Alain Martelli1,2,3,4,5, Florent Colin1,2,3,4,5, Adeline Page1,2,3,4, Marie Wattenhofer-Donzé1,2,3,4,5, Laurence Reutenauer1,2,3,4,5, Hélène Puccio1,2,3,4,5*
1 Department of Translational Medicine and Neurogenetics, Institut de Génétique et de Biologie Moléculaire et Cellulaire (IGBMC), Illkirch, France, 2 Inserm U596, Illkirch, France, 3 CNRS UMR7104, Illkirch, France, 4 Université de Strasbourg, Strasbourg, France, 5 Chaire de Génétique Humaine, Collège de France, Illkirch, France
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Conclusions/Significance
Our results suggest that the interaction of frataxin with the core ISCU/NFS1/ISD11 complex most likely defines the essential function of frataxin. Our results provide new elements important for further understanding the early steps of de novo Fe-S cluster biosynthesis
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Stéphane Schmucker1,2,3,4,5, Alain Martelli1,2,3,4,5, Florent Colin1,2,3,4,5, Adeline Page1,2,3,4, Marie Wattenhofer-Donzé1,2,3,4,5, Laurence Reutenauer1,2,3,4,5, Hélène Puccio1,2,3,4,5*
1 Department of Translational Medicine and Neurogenetics, Institut de Génétique et de Biologie Moléculaire et Cellulaire (IGBMC), Illkirch, France, 2 Inserm U596, Illkirch, France, 3 CNRS UMR7104, Illkirch, France, 4 Université de Strasbourg, Strasbourg, France, 5 Chaire de Génétique Humaine, Collège de France, Illkirch, France
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Conclusions/Significance
Our results suggest that the interaction of frataxin with the core ISCU/NFS1/ISD11 complex most likely defines the essential function of frataxin. Our results provide new elements important for further understanding the early steps of de novo Fe-S cluster biosynthesis
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The Mitochondrial Connection in Auditory Neuropathy.
Audiol Neurotol 2011;16:398-413 (DOI: 10.1159/000323276)
Cacace AT, Pinheiro JM.
Department of Communication Sciences and Disorders, Wayne State University, Detroit, Mich., USA.
Keywords: Auditory brainstem responses, Auditory neuropathy, Charcot-Marie-Tooth disease, Autosomal dominant optic atrophy, Cochlear microphonics, Friedreich’s ataxia, Hyperbilirubinemia, Respiratory chain, Leber’s hereditary optic neuropathy, Mitochondria, Otoacoustic emissions.
Cacace AT, Pinheiro JM.
Department of Communication Sciences and Disorders, Wayne State University, Detroit, Mich., USA.
Keywords: Auditory brainstem responses, Auditory neuropathy, Charcot-Marie-Tooth disease, Autosomal dominant optic atrophy, Cochlear microphonics, Friedreich’s ataxia, Hyperbilirubinemia, Respiratory chain, Leber’s hereditary optic neuropathy, Mitochondria, Otoacoustic emissions.
Analysis of nucleosome positioning determined by DNA helix curvature in the human genome
Hongde Liu, Xueye Duan, Shuangxin Yu and Xiao Sun.
BMC Genomics 2011, 12:72doi:10.1186/1471-2164-12-72
Published: 27 January 2011
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BMC Genomics 2011, 12:72doi:10.1186/1471-2164-12-72
Published: 27 January 2011
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