Medical News Today, Article Date: 22 Feb 2011
"complement of trials being offered at UHealth broadens the availability of experimental therapy to patients and offers an alternative to the growing trend of medical tourism for cell based therapies." (University of Miami Miller School of Medicine) Read more ...
Wednesday, February 23, 2011
Monday, February 21, 2011
Neuronal Activity Controls the Antagonistic Balance Between Peroxisome Proliferator-Activated Receptor-γ Coactivator-1α and Silencing Mediator of Retinoic Acid and Thyroid Hormone Receptors in Regulating Antioxidant Defenses
Antioxidants & Redox Signaling (14, 000–000). -Online Ahead of Print: February 20, 2011-. doi:10.1089/ars.2010.3568.
Francesc X. Soriano, Frédéric Léveillé, Sofia Papadia, Karen F.S. Bell, Clare Puddifoot, Giles E. Hardingham.
Centre for Integrative Physiology, University of Edinburgh
Keywords: Transcriptional coactivators and corepressors, multiple targets, coactivator peroxisome proliferator-activated receptor-γ coactivator (PGC)-1α, Huntington's, antioxidant defenses, mitochondrial biogenesis, retinoic acid and thyroid hormone receptors (SMRT), implications for neuronal viability.
Francesc X. Soriano, Frédéric Léveillé, Sofia Papadia, Karen F.S. Bell, Clare Puddifoot, Giles E. Hardingham.
Centre for Integrative Physiology, University of Edinburgh
Keywords: Transcriptional coactivators and corepressors, multiple targets, coactivator peroxisome proliferator-activated receptor-γ coactivator (PGC)-1α, Huntington's, antioxidant defenses, mitochondrial biogenesis, retinoic acid and thyroid hormone receptors (SMRT), implications for neuronal viability.
Saturday, February 19, 2011
Transition to adult care for children with chronic neurological disorders
Annals of Neurology, 2011, DOI: 10.1002/ana.22393
Peter Camfield MD, Carol Camfield MD
"We make a series of suggestions about how to improve the transition/transfer process with the hope of better medical and social adult outcome for children with neurological disorders."
Peter Camfield MD, Carol Camfield MD
"We make a series of suggestions about how to improve the transition/transfer process with the hope of better medical and social adult outcome for children with neurological disorders."
Friday, February 18, 2011
Quantification of Circulating Plasma DNA in Friedreich's Ataxia and Spinocerebellar Ataxia Types 2 and 12
DNA and Cell Biology. null, Vol. 0, No. 0, Online Ahead of Print: February 17, 2011. doi:10.1089/dna.2010.1165.
Vishnu Swarup, Achal K. Srivastava, Madakasira V. Padma, Moganty R. Rajeswari.
Department of Biochemistry, All India Institute of Medical Sciences, New Delhi, India.
Department of Neurology, All India Institute of Medical Sciences, New Delhi, India.
Keywords: DNA triplet repeat expansion, Friedreich's ataxia, spinocerebellar ataxias (SCAs), multisystem neurodegenerative disorders, Cell-free circulating DNA in plasma, clinical diagnosis, International Co-operative Ataxia Rating Scale, plasma DNA, PicoGreen fluorescent assay, neuronal and muscular degeneration.
Vishnu Swarup, Achal K. Srivastava, Madakasira V. Padma, Moganty R. Rajeswari.
Department of Biochemistry, All India Institute of Medical Sciences, New Delhi, India.
Department of Neurology, All India Institute of Medical Sciences, New Delhi, India.
Keywords: DNA triplet repeat expansion, Friedreich's ataxia, spinocerebellar ataxias (SCAs), multisystem neurodegenerative disorders, Cell-free circulating DNA in plasma, clinical diagnosis, International Co-operative Ataxia Rating Scale, plasma DNA, PicoGreen fluorescent assay, neuronal and muscular degeneration.
Friday, February 11, 2011
Friedreich's ataxia: Pathology, pathogenesis, and molecular genetics
Journal of the Neurological Sciences, doi:10.1016/j.jns.2011.01.010
Arnulf H. Koeppen
VA Medical Center, 113 Holland Ave, and Albany Medical College, 47 New Scotland Ave, Albany, NY 12208 USA, Available online 10 February 2011.
Keywords: Cardiomyopathy; Dentate nucleus; Dorsal root ganglion; Frataxin; Friedreich's ataxia; GAA trinucleotide repeats; Iron; Transcription
Arnulf H. Koeppen
VA Medical Center, 113 Holland Ave, and Albany Medical College, 47 New Scotland Ave, Albany, NY 12208 USA, Available online 10 February 2011.
Keywords: Cardiomyopathy; Dentate nucleus; Dorsal root ganglion; Frataxin; Friedreich's ataxia; GAA trinucleotide repeats; Iron; Transcription
Tuesday, February 8, 2011
Gene therapy finds its niche
Nature Biotechnology Volume: 29, Pages: 121–128 Year published:(2011)
doi:10.1038/nbt.1769
Cormac Sheridan
Gene therapy is finally poised to make a contribution to the treatment of debilitating, highly penetrant genetic diseases that have proved intractable to other regimens.
doi:10.1038/nbt.1769
Cormac Sheridan
Gene therapy is finally poised to make a contribution to the treatment of debilitating, highly penetrant genetic diseases that have proved intractable to other regimens.
ReNeuron and StemCells get green light for neural stem cell trials
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.
George S Mack
Maybe not as fast as we would like, but progress is being made in stem cell therapies for neurological diseases.
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.
FREE ACCESS
"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.
FREE ACCESS
"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."
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