Research Topics
Species | Dinah W Y SahSummaryAffiliation: Alnylam Pharmaceuticals Country: USA Publications
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Detail Information
Publications
Therapeutic potential of RNA interference for neurological disordersDinah W Y Sah
Alnylam Pharmaceuticals Inc, Cambridge, MA 02142, USA
Life Sci 79:1773-80. 2006....
A status report on RNAi therapeuticsAkshay K Vaishnaw
Alnylam Pharmaceuticals Inc, 300 Third Street, Cambridge, MA 02142, USA
Silence 1:14. 2010....- Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanismsAkin Akinc
Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA
Mol Ther 18:1357-64. 2010..Both apoE-based endogenous and GalNAc-based exogenous targeting appear to be highly effective strategies for the delivery of iLNPs to liver... 
Direct CNS delivery of siRNA mediates robust silencing in oligodendrocytesWilliam Querbes
Alnylam Pharmaceuticals, Inc, Cambridge, Massachusetts 02142, USA
Oligonucleotides 19:23-29. 2009..Taken together, these results show for the first time robust RNAi within oligodendrocytes in vivo and demonstrate the important potential of siRNAs in the treatment of CNS disorders involving oligodendrocyte pathology...- Oligonucleotide therapeutic approaches for Huntington diseaseDinah W Y Sah
Alnylam Pharmaceuticals Inc, Cambridge, Massachusetts, USA
J Clin Invest 121:500-7. 2011..Delivery remains a key challenge for translational success, especially with chronic therapy. The potential of disease-modifying oligonucleotide approaches for Huntington disease will be revealed as they progress into clinical trials... - Lipophilic siRNAs mediate efficient gene silencing in oligodendrocytes with direct CNS deliveryQingmin Chen
Alnylam Pharmaceuticals Inc, 300 Third Street, Cambridge, MA 02142, USA
J Control Release 144:227-32. 2010.... - Lipid-like materials for low-dose, in vivo gene silencingKevin T Love
Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA
Proc Natl Acad Sci U S A 107:1864-9. 2010..03 mg/kg. To our knowledge, this formulation facilitates gene silencing at orders-of-magnitude lower doses than required by any previously described siRNA liver delivery system... - Novel therapeutic modalities to address nondrugable protein interaction targetsErrol B De Souza
Archemix Corporation, Cambridge, MA 02142, USA
Neuropsychopharmacology 34:142-58. 2009..Finally, examples of their application as therapeutics for the treatment of pain and some neurological disorders such as Alzheimer's disease, multiple sclerosis, Huntington's disease, and Parkinson's disease are provided... - A combinatorial library of lipid-like materials for delivery of RNAi therapeuticsAkin Akinc
David H Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, Massachusetts 02139, USA
Nat Biotechnol 26:561-9. 2008..The studies reported here suggest that these materials may have broad utility for both local and systemic delivery of RNA therapeutics... - RNAi therapeutics: a potential new class of pharmaceutical drugsDavid Bumcrot
Alnylam Pharmaceuticals Inc, 300 Third Street, Cambridge, MA 02142, USA
Nat Chem Biol 2:711-9. 2006..With advances in this area and the commencement of multiple clinical trials with RNAi therapeutic candidates, a transformation in modern medicine may soon be realized... - Distribution of GDNF family receptor alpha3 and RET in rat and human non-neural tissuesChunhua Yang
BiogenIdec, Inc, 14 Cambridge Center, Cambridge, MA 02142, USA
J Mol Histol 37:69-77. 2006..In other tissues, sub-populations of cells expressed either GFRalpha3- or RET-like immunoreactivity. The functional consequences of GFRalpha3 expression in non-neural cells remain to be determined... - New approaches for the treatment of pain: the GDNF family of neurotrophic growth factorsDinah W Y Sah
Alnylam Pharmaceuticals, Inc, 300 Third Street, Cambridge, MA 02142, USA
Curr Top Med Chem 5:577-83. 2005..Structural considerations, particularly with regard to implications for binding interactions and biological activity are discussed... - siRNA knock-down of mutant torsinA restores processing through secretory pathway in DYT1 dystonia cellsJeffrey W Hewett
Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA 02129, USA
Hum Mol Genet 17:1436-45. 2008..The ability of allele-specific siRNA for torsinADeltaE to normalize secretory function in DYT1 patient cells supports its potential role as a therapeutic agent in early onset torsion dystonia...