Dr Seng H Cheng, PhD - SANOFI - Global Head of Research, Rare Disease

Dr Seng H Cheng

PhD

SANOFI

Global Head of Research, Rare Disease

Framingham, MA | United States

Dr Seng H Cheng, PhD - SANOFI - Global Head of Research, Rare Disease

Dr Seng H Cheng

PhD

Introduction

Primary Affiliation: SANOFI - Framingham, MA , United States

Publications

99Publications

4273Reads

3Profile Views

2242PubMed Central Citations

Molecular basis for globotriaosylceramide regulation and enzyme uptake in immortalized aortic endothelial cells from Fabry mice.

J Inherit Metab Dis 2016 05 10;39(3):447-455. Epub 2016 Mar 10.

Institute of Metabolic Disease, Baylor Research Institute, 3812 Elm Street, Dallas, TX, 75226, USA.

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http://dx.doi.org/10.1007/s10545-016-9920-6DOI Listing
May 2016
23 Reads
3.370 Impact Factor

AAV8-mediated expression of N-acetylglucosamine-1-phosphate transferase attenuates bone loss in a mouse model of mucolipidosis II.

Mol Genet Metab 2016 Apr 3;117(4):447-55. Epub 2016 Feb 3.

Department of Medical Genetics, Ajou University Hospital, Ajou University School of Medicine, Suwon, Republic of Korea.

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http://dx.doi.org/10.1016/j.ymgme.2016.02.001DOI Listing
April 2016
36 Reads
1 Citation
2.625 Impact Factor

Fibrillin-1 Regulates Skeletal Stem Cell Differentiation by Modulating TGFβ Activity Within the Marrow Niche.

J Bone Miner Res 2016 Jan 17;31(1):86-97. Epub 2015 Aug 17.

Department of Pharmacology and Systems Therapeutics, Icahn School of Medicine at Mount Sinai, New York, NY, USA.

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http://dx.doi.org/10.1002/jbmr.2598DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5776390PMC
January 2016
27 Reads
9 Citations
6.832 Impact Factor

Analytical Ultracentrifugation as an Approach to Characterize Recombinant Adeno-Associated Viral Vectors.

Hum Gene Ther Methods 2015 Dec 15;26(6):228-42. Epub 2015 Oct 15.

Gene Therapy, Genzyme, a Sanofi Company , Framingham, Massachusetts.

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http://dx.doi.org/10.1089/hgtb.2015.048DOI Listing
December 2015
19 Reads
6 Citations
1.641 Impact Factor

Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial.

September 2015
74 Reads
44 Citations

Glycosphingolipids are modulators of disease pathogenesis in amyotrophic lateral sclerosis.

Proc Natl Acad Sci U S A 2015 Jun 8;112(26):8100-5. Epub 2015 Jun 8.

Rare Diseases Science, Genzyme, a Sanofi Company, Framingham, MA 01701;

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http://dx.doi.org/10.1073/pnas.1508767112DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4491749PMC
June 2015
78 Reads
9 Citations
9.810 Impact Factor

Dimorphic effects of transforming growth factor-β signaling during aortic aneurysm progression in mice suggest a combinatorial therapy for Marfan syndrome.

Arterioscler Thromb Vasc Biol 2015 Apr 22;35(4):911-7. Epub 2015 Jan 22.

From the Department of Pharmacology and Systems Therapeutics, Icahn School of Medicine at Mount Sinai, New York, NY (J.R.C., L.C., J.G., E.C., S.S., F.R.); and Genzyme, a Sanofi Company, Framingham, MA (N.P.C., C.A.N., S.H.C., B.M.W.).

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http://dx.doi.org/10.1161/ATVBAHA.114.305150DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4376614PMC
April 2015
44 Reads
45 Citations
6.000 Impact Factor

α-Galactosidase A knockout mice: progressive organ pathology resembles the type 2 later-onset phenotype of Fabry disease.

Am J Pathol 2015 Mar 29;185(3):651-65. Epub 2014 Dec 29.

Department of Pathology, Genzyme Corporation, a Sanofi Company, Framingham, Massachusetts.

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http://dx.doi.org/10.1016/j.ajpath.2014.11.004DOI Listing
March 2015
73 Reads
9 Citations
4.591 Impact Factor

Gaucher-related synucleinopathies: the examination of sporadic neurodegeneration from a rare (disease) angle.

Prog Neurobiol 2015 Feb 6;125:47-62. Epub 2015 Jan 6.

Genzyme, a Sanofi Company, 49 New York Avenue, Framingham, MA 01701, USA.

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http://dx.doi.org/10.1016/j.pneurobio.2014.12.001DOI Listing
February 2015
139 Reads
13 Citations
9.992 Impact Factor

Gene therapy for the neurological manifestations in lysosomal storage disorders.

Authors:
Seng H Cheng

J Lipid Res 2014 Sep 29;55(9):1827-38. Epub 2014 Mar 29.

Genzyme, a Sanofi Company, Framingham, MA 01701-9322.

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http://dx.doi.org/10.1194/jlr.R047175DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4617364PMC
September 2014
38 Reads
8 Citations
4.421 Impact Factor

Reversibility of neuropathology in Tay-Sachs-related diseases.

Hum Mol Genet 2014 Feb 20;23(3):730-48. Epub 2013 Sep 20.

Department of Medicine, University of Cambridge, Cambridge, UK and.

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http://dx.doi.org/10.1093/hmg/ddt459DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3888261PMC
February 2014
74 Reads
10 Citations
6.393 Impact Factor

Gene therapy for lysosomal storage disorders.

Pediatr Endocrinol Rev 2013 Nov;11 Suppl 1:99-109

Genzyme, Sanofi Company, 49 New York Avenue, Framingham, MA 01701-9322, USA.

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November 2013
44 Reads
2 Citations

Dysregulation of multiple facets of glycogen metabolism in a murine model of Pompe disease.

PLoS One 2013 14;8(2):e56181. Epub 2013 Feb 14.

Genzyme, a Sanofi Company, Framingham, Massachusetts, United States of America.

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http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0056181PLOS
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3572993PMC
August 2013
39 Reads
7 Citations
3.234 Impact Factor

Erythrocytes encapsulated with phenylalanine hydroxylase exhibit improved pharmacokinetics and lowered plasma phenylalanine levels in normal mice.

Mol Genet Metab 2013 Aug 16;109(4):339-44. Epub 2013 Jul 16.

Genzyme, a Sanofi Company, 49 New York Avenue, Framingham, MA 01701-9322, USA.

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http://dx.doi.org/10.1016/j.ymgme.2013.05.011DOI Listing
August 2013
29 Reads
1 Citation
2.625 Impact Factor

Systemic delivery of a Peptide-linked morpholino oligonucleotide neutralizes mutant RNA toxicity in a mouse model of myotonic dystrophy.

Nucleic Acid Ther 2013 Apr 11;23(2):109-17. Epub 2013 Jan 11.

Genzyme, a Sanofi Company , Framingham, MA 01701, USA.

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http://dx.doi.org/10.1089/nat.2012.0404DOI Listing
April 2013
37 Reads
16 Citations
2.890 Impact Factor

Rapid identification of novel functional promoters for gene therapy.

J Mol Med (Berl) 2012 Dec 6;90(12):1487-96. Epub 2012 Jul 6.

GeneMedicine Research Group, NDCLS, John Radcliffe Hospital, University of Oxford, OX3 9DU Oxford, UK.

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http://dx.doi.org/10.1007/s00109-012-0928-6DOI Listing
December 2012
42 Reads
3 Citations
5.110 Impact Factor

Merits of combination cortical, subcortical, and cerebellar injections for the treatment of Niemann-Pick disease type A.

Mol Ther 2012 Oct 24;20(10):1893-901. Epub 2012 Jul 24.

Rare Diseases Division, Genzyme Corporation, Framingham, Massachusetts 01701-9322, USA.

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http://dx.doi.org/10.1038/mt.2012.118DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3464981PMC
October 2012
54 Reads
10 Citations
6.230 Impact Factor

Gene transfer corrects acute GM2 gangliosidosis--potential therapeutic contribution of perivascular enzyme flow.

Mol Ther 2012 Aug 27;20(8):1489-500. Epub 2012 Mar 27.

Department of Medicine, University of Cambridge, Cambridge, UK.

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http://dx.doi.org/10.1038/mt.2012.44DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3412498PMC
August 2012
66 Reads
18 Citations
6.230 Impact Factor

Proteasome inhibition is partially effective in attenuating pre-existing immunity against recombinant adeno-associated viral vectors.

PLoS One 2012 13;7(4):e34684. Epub 2012 Apr 13.

Genetic Disease Science, Genzyme Corporation, Framingham, Massachusetts, United States of America.

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http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0034684PLOS
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3326043PMC
August 2012
7 Reads
5 Citations
3.234 Impact Factor

Mutant GBA1 expression and synucleinopathy risk: first insights from cellular and mouse models.

Neurodegener Dis 2012 9;10(1-4):195-202. Epub 2012 Feb 9.

Genzyme, Sanofi Company, Framingham, Mass 01701, USA.

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http://dx.doi.org/10.1159/000335038DOI Listing
August 2012
9 Reads
9 Citations
3.511 Impact Factor

Assessment of the nuclear pore dilating agent trans-cyclohexane-1,2-diol in differentiated airway epithelium.

J Gene Med 2012 Jul;14(7):491-500

Department of Gene Therapy, Imperial College London, UK.

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http://dx.doi.org/10.1002/jgm.2643DOI Listing
July 2012
46 Reads
3 Citations
2.472 Impact Factor

Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis.

Neuron 2012 Jun;74(6):1031-44

Ludwig Institute for Cancer Research and Department of Cellular and Molecular Medicine, University of California at San Diego, 9500 Gilman Drive, La Jolla, CA 92093, USA.

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http://dx.doi.org/10.1016/j.neuron.2012.05.009DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3383626PMC
June 2012
129 Reads
192 Citations
15.054 Impact Factor

Marked differences in neurochemistry and aggregates despite similar behavioural and neuropathological features of Huntington disease in the full-length BACHD and YAC128 mice.

Hum Mol Genet 2012 May 9;21(10):2219-32. Epub 2012 Feb 9.

Centre for Molecular Medicine and Therapeutics, University of British Columbia, and Child and Family Research Institute, Vancouver, BC, Canada.

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http://dx.doi.org/10.1093/hmg/dds037DOI Listing
May 2012
19 Reads
44 Citations
6.393 Impact Factor

IGF-1 delivery to CNS attenuates motor neuron cell death but does not improve motor function in type III SMA mice.

Neurobiol Dis 2012 Jan 18;45(1):272-9. Epub 2011 Aug 18.

Department of Neurology, National Taiwan University Hospital and National Taiwan University College of Medicine, Taipei 100, Taiwan.

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http://dx.doi.org/10.1016/j.nbd.2011.06.021DOI Listing
January 2012
12 Reads
16 Citations
5.080 Impact Factor

Disease progression in a mouse model of amyotrophic lateral sclerosis: the influence of chronic stress and corticosterone.

FASEB J 2011 Dec 29;25(12):4369-77. Epub 2011 Aug 29.

Genzyme Corporation, 49 New York Ave., Framingham, MA 01701-9322, USA.

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http://dx.doi.org/10.1096/fj.11-190819DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3236626PMC
December 2011
4 Reads
8 Citations
5.043 Impact Factor

Adeno-associated virus-mediated expression of β-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain.

Hum Mol Genet 2011 Nov 18;20(22):4371-80. Epub 2011 Aug 18.

Department of Medicine, University of Cambridge, Level 5 Addenbrooke's Hospital, Box 157, Hills Road, Cambridge CB2 0QQ, UK.

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http://dx.doi.org/10.1093/hmg/ddr364DOI Listing
November 2011
17 Reads
18 Citations
6.393 Impact Factor

Neural stem cell transplantation as a therapeutic approach for treating lysosomal storage diseases.

Neurotherapeutics 2011 Oct;8(4):659-67

Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701-9322, USA.

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http://dx.doi.org/10.1007/s13311-011-0067-8DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3250293PMC
October 2011
8 Reads
5 Citations

Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system.

FASEB J 2011 Oct 11;25(10):3505-18. Epub 2011 Jul 11.

Gene Transfer Technology Group, Institute for Women's Health, University College London, 86-96 Chenies Mews, London, WC1E 6HX, UK.

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http://dx.doi.org/10.1096/fj.11-182311DOI Listing
October 2011
18 Reads
33 Citations
5.043 Impact Factor

Acid β-glucosidase mutants linked to Gaucher disease, Parkinson disease, and Lewy body dementia alter α-synuclein processing.

Ann Neurol 2011 Jun 6;69(6):940-53. Epub 2011 Apr 6.

Center for Neurologic Diseases, Department of Neurology, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA.

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http://dx.doi.org/10.1002/ana.22400DOI Listing
June 2011
82 Reads
95 Citations
9.980 Impact Factor

Prospects for the gene therapy of spinal muscular atrophy.

Trends Mol Med 2011 May 19;17(5):259-65. Epub 2011 Feb 19.

Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701-9322, USA.

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http://dx.doi.org/10.1016/j.molmed.2011.01.002DOI Listing
May 2011
46 Reads
9 Citations
9.453 Impact Factor

Relationship between neuropathology and disease progression in the SOD1(G93A) ALS mouse.

Exp Neurol 2011 Feb 9;227(2):287-95. Epub 2010 Dec 9.

Genzyme Corporation, 49 New York Ave, Framingham, MA 01701-9322, USA.

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http://dx.doi.org/10.1016/j.expneurol.2010.11.019DOI Listing
February 2011
33 Reads
13 Citations
4.700 Impact Factor

Induction of immune tolerance to a therapeutic protein by intrathymic gene delivery.

Mol Ther 2010 Dec 27;18(12):2146-54. Epub 2010 Jul 27.

Genzyme Corporation, Framingham, Massachusetts 01701, USA.

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http://dx.doi.org/10.1038/mt.2010.164DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2997582PMC
December 2010
51 Reads
6 Citations
6.230 Impact Factor

Inhibition of osteoclastogenesis by prolyl hydroxylase inhibitor dimethyloxallyl glycine.

J Bone Miner Metab 2010 Sep 19;28(5):510-9. Epub 2010 Mar 19.

Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701-9322, USA.

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http://dx.doi.org/10.1007/s00774-010-0171-6DOI Listing
September 2010
40 Reads
7 Citations
2.460 Impact Factor

Evaluation of systemic follistatin as an adjuvant to stimulate muscle repair and improve motor function in Pompe mice.

Mol Ther 2010 Sep 15;18(9):1584-91. Epub 2010 Jun 15.

Genzyme Corporation, Framingham, Massachusetts, USA.

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http://dx.doi.org/10.1038/mt.2010.110DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2956929PMC
September 2010
31 Reads
6 Citations
6.230 Impact Factor

Reducing glycosphingolipid biosynthesis in airway cells partially ameliorates disease manifestations in a mouse model of asthma.

Int Immunol 2010 Jul 24;22(7):593-603. Epub 2010 May 24.

Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701, USA.

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http://dx.doi.org/10.1093/intimm/dxq044DOI Listing
July 2010
14 Reads
6 Citations
2.540 Impact Factor

CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy.

J Clin Invest 2010 Apr 15;120(4):1253-64. Epub 2010 Mar 15.

Genzyme Corporation, 49 New York Avenue, Room 2410, Framingham, MA 01701, USA.

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http://dx.doi.org/10.1172/JCI41615DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2846065PMC
April 2010
57 Reads
116 Citations
13.215 Impact Factor

Low-frequency ultrasound increases non-viral gene transfer to the mouse lung.

Acta Biochim Biophys Sin (Shanghai) 2010 Jan;42(1):45-51

Department of Gene Therapy, National Heart and Lung Institute, Faculty of Medicine, Imperial College, London, UK.

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http://dx.doi.org/10.1093/abbs/gmp100DOI Listing
January 2010
37 Reads
2 Citations
2.191 Impact Factor

AAV8-mediated gene therapy prevents induced biochemical attacks of acute intermittent porphyria and improves neuromotor function.

Mol Ther 2010 Jan 27;18(1):17-22. Epub 2009 Oct 27.

Department of Genetics and Genomic Sciences, Mount Sinai School of Medicine, New York, New York, USA.

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http://dx.doi.org/10.1038/mt.2009.250DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2839207PMC
January 2010
9 Reads
7 Citations
6.230 Impact Factor

Detection of CFTR transgene mRNA expression in respiratory epithelium isolated from the murine nasal cavity.

J Gene Med 2010 Jan;12(1):55-63

Medical Genetics Section, Molecular Medicine Centre, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK.

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http://dx.doi.org/10.1002/jgm.1413DOI Listing
January 2010
27 Reads
2 Citations
2.472 Impact Factor

The role of doxorubicin in non-viral gene transfer in the lung.

Biomaterials 2009 Apr 18;30(10):1971-7. Epub 2009 Jan 18.

Department of Gene Therapy, Imperial College at the National Heart and Lung Institute, Manresa Road, London SW36LR, UK.

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http://dx.doi.org/10.1016/j.biomaterials.2008.12.037DOI Listing
April 2009
48 Reads
3 Citations
8.560 Impact Factor

Temporal neuropathologic and behavioral phenotype of 6neo/6neo Pompe disease mice.

J Neuropathol Exp Neurol 2008 Aug;67(8):803-18

Department of Neurology (RLS), Beth Israel Deaconess Medical Center, Boston, Massachusetts, USA.

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http://dx.doi.org/10.1097/NEN.0b013e3181815994DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2743262PMC
August 2008
28 Reads
28 Citations
3.800 Impact Factor

CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression.

Nat Biotechnol 2008 May 27;26(5):549-51. Epub 2008 Apr 27.

Gene Medicine Research Group, Nuffield Department of Clinical Laboratory Sciences, University of Oxford, Oxford OX3 9DU, UK.

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http://dx.doi.org/10.1038/nbt1399DOI Listing
May 2008
52 Reads
61 Citations
41.514 Impact Factor

Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis.

Mol Ther 2008 Apr 12;16(4):649-56. Epub 2008 Feb 12.

Department of Molecular Physiology and Biophysics, University of Iowa, Iowa City, Iowa 52242, USA.

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http://dx.doi.org/10.1038/mt.2008.9DOI Listing
April 2008
43 Reads
56 Citations
6.230 Impact Factor

In vivo imaging of gene transfer to the respiratory tract.

Biomaterials 2008 Apr 21;29(10):1533-40. Epub 2007 Dec 21.

Department of Gene Therapy, Imperial College at the National Heart and Lung Institute, Manresa Road, London SW3 6LR, UK.

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http://dx.doi.org/10.1016/j.biomaterials.2007.11.017DOI Listing
April 2008
41 Reads
4 Citations
8.560 Impact Factor

Hypoxia-inducible factor 1 mediates hypoxia-induced cardiomyocyte lipid accumulation by reducing the DNA binding activity of peroxisome proliferator-activated receptor alpha/retinoid X receptor.

Biochem Biophys Res Commun 2007 Dec 22;364(3):567-72. Epub 2007 Oct 22.

Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701, USA.

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http://dx.doi.org/10.1016/j.bbrc.2007.10.062DOI Listing
December 2007
5 Reads
15 Citations
2.300 Impact Factor

Intraparenchymal injections of acid sphingomyelinase results in regional correction of lysosomal storage pathology in the Niemann-Pick A mouse.

Exp Neurol 2007 Oct 10;207(2):258-66. Epub 2007 Jul 10.

Genzyme Corporation, One Mountain Road, Framingham, MA 01701-9322, USA.

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http://dx.doi.org/10.1016/j.expneurol.2007.06.017DOI Listing
October 2007
21 Reads
3 Citations
4.700 Impact Factor

Acute intermittent porphyria: vector optimization for gene therapy.

J Gene Med 2007 Sep;9(9):806-11

Department of Genetics and Genomic Sciences, Mount Sinai School of Medicine, New York, NY 10029, USA.

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http://dx.doi.org/10.1002/jgm.1074DOI Listing
September 2007
37 Reads
1 Citation
2.472 Impact Factor

Top co-authors

Lamya S Shihabuddin
30
Ronald K Scheule
26
James C Dodge
James C Dodge

Genzyme Corporation

19
Marco A Passini
Marco A Passini

Genzyme Corporation

17
Christopher M Treleaven
Christopher M Treleaven

Rare Diseases

13
John Marshall
John Marshall

University of Toronto

12
Uta Griesenbach
Uta Griesenbach

Imperial College London

12
Canwen Jiang
Canwen Jiang

Genzyme Corporation

11
Deborah R Gill
Deborah R Gill

University of Oxford

10