Dwight D Koeberl

Dwight D Koeberl

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Dwight D Koeberl

Publications by authors named "Dwight D Koeberl"

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Gene therapy for glycogen storage diseases.

Hum Mol Genet 2019 Oct;28(R1):R31-R41

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

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http://dx.doi.org/10.1093/hmg/ddz133DOI Listing
October 2019

Salmeterol with Liver Depot Gene Therapy Enhances the Skeletal Muscle Response in Murine Pompe Disease.

Hum Gene Ther 2019 Jul 5;30(7):855-864. Epub 2019 Apr 5.

1Division of Medical Genetics, Department of Pediatrics, Duke University Medical School, Durham, North Carolina.

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http://dx.doi.org/10.1089/hum.2018.197DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6648189PMC
July 2019

Liver depot gene therapy for Pompe disease.

Ann Transl Med 2019 Jul;7(13):288

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USA.

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http://dx.doi.org/10.21037/atm.2019.05.02DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6642935PMC
July 2019

Bezafibrate Enhances AAV Vector-Mediated Genome Editing in Glycogen Storage Disease Type Ia.

Mol Ther Methods Clin Dev 2019 Jun 10;13:265-273. Epub 2019 Feb 10.

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

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http://dx.doi.org/10.1016/j.omtm.2019.02.002DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6395830PMC
June 2019

Letter to the Editors: Concerning "Long-term safety and efficacy of AAV gene therapy in the canine model of glycogen storage disease type Ia" by Lee et al.

J Inherit Metab Dis 2018 11 25;41(6):913-914. Epub 2018 Sep 25.

Division of Medical Genetics, Duke University Medical Center, DUMC Box 103856, Durham, NC, 27710, USA.

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http://link.springer.com/10.1007/s10545-018-0248-2
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http://dx.doi.org/10.1007/s10545-018-0248-2DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6501822PMC
November 2018

Treatment outcome of twenty-two patients with guanidinoacetate methyltransferase deficiency: An international retrospective cohort study.

Eur J Paediatr Neurol 2018 May 16;22(3):369-379. Epub 2018 Feb 16.

Genetics and Genome Biology Program, Research Institute, The Hospital for Sick Children, Toronto, ON, Canada; Division of Clinical and Metabolic Genetics, Department of Paediatrics, University of Toronto, The Hospital for Sick Children, Toronto, ON, Canada. Electronic address:

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http://dx.doi.org/10.1016/j.ejpn.2018.02.007DOI Listing
May 2018

Renal endoplasmic reticulum stress is coupled to impaired autophagy in a mouse model of GSD Ia.

Mol Genet Metab 2017 11 1;122(3):95-98. Epub 2017 Sep 1.

Cardiovascular and Metabolic Disorders Program, Duke-NUS Medical School, Singapore, Singapore; Duke Molecular Physiology Institute, Duke University School of Medicine, Durham, NC, USA. Electronic address:

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http://dx.doi.org/10.1016/j.ymgme.2017.08.013DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5722666PMC
November 2017

Hepatic mitochondrial dysfunction is a feature of Glycogen Storage Disease Type Ia (GSDIa).

Sci Rep 2017 03 20;7:44408. Epub 2017 Mar 20.

Cardiovascular and Metabolic Disorders Program, Duke-NUS Medical School Singapore, Singapore.

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http://dx.doi.org/10.1038/srep44408DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5357851PMC
March 2017

Preclinical Development of New Therapy for Glycogen Storage Diseases.

Curr Gene Ther 2015 ;15(4):338-47

Box 103856, Duke University Medical Center, Durham, NC 27710.

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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4682885PMC
June 2016

Vision of correction for classic homocystinuria.

Authors:
Dwight D Koeberl

J Clin Invest 2016 06 16;126(6):2043-4. Epub 2016 May 16.

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http://dx.doi.org/10.1172/JCI88251DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4887184PMC
June 2016

Salmeterol enhances the cardiac response to gene therapy in Pompe disease.

Mol Genet Metab 2016 May 18;118(1):35-40. Epub 2016 Mar 18.

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, United States.

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http://dx.doi.org/10.1016/j.ymgme.2016.03.006DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4833676PMC
May 2016

A beta-blocker, propranolol, decreases the efficacy from enzyme replacement therapy in Pompe disease.

Mol Genet Metab 2016 Feb 3;117(2):114-9. Epub 2015 Oct 3.

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, United States.

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http://dx.doi.org/10.1016/j.ymgme.2015.09.012DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4755835PMC
February 2016

Induction of autophagy improves hepatic lipid metabolism in glucose-6-phosphatase deficiency.

J Hepatol 2016 Feb 20;64(2):370-379. Epub 2015 Oct 20.

Cardiovascular and Metabolic Disorders Program, Duke-NUS Graduate Medical School Singapore, Singapore; Sarah W. Stedman Nutrition and Metabolism Center, Departments of Medicine and Pharmacology and Cancer Biology, Duke University Medical Center, Durham, NC, USA. Electronic address:

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http://dx.doi.org/10.1016/j.jhep.2015.10.008DOI Listing
February 2016

Complex III deficiency due to an in-frame MT-CYB deletion presenting as ketotic hypoglycemia and lactic acidosis.

Mol Genet Metab Rep 2015 Sep 30;4:39-41. Epub 2015 Jun 30.

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USA; Biochemical Genetics Laboratory, Duke University Medical Center, Durham, NC, USA.

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http://dx.doi.org/10.1016/j.ymgmr.2015.06.001DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4750615PMC
September 2015

Assessment of toxicity and biodistribution of recombinant AAV8 vector-mediated immunomodulatory gene therapy in mice with Pompe disease.

Mol Ther Methods Clin Dev 2014 11;1:14018. Epub 2014 Jun 11.

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center , Durham, North Carolina, USA.

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http://dx.doi.org/10.1038/mtm.2014.18DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4362383PMC
May 2015

Large animal models and new therapies for glycogen storage disease.

J Inherit Metab Dis 2015 May 16;38(3):505-9. Epub 2014 Sep 16.

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USA.

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http://dx.doi.org/10.1007/s10545-014-9766-8DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4513910PMC
May 2015

A natural choice for hemophilia B.

Authors:
Dwight D Koeberl

Blood 2015 Mar;125(10):1509-10

DUKE UNIVERSITY MEDICAL CENTER; DEPARTMENT OF MOLECULAR GENETICS AND MICROBIOLOGY, DUKE UNIVERSITY.

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http://dx.doi.org/10.1182/blood-2015-01-622506DOI Listing
March 2015

The heart is just a muscle.

Circulation 2015 Mar;131(10):914-22

From Division of Cardiology, Department of Medicine (R.W.M., T.A., C.B.P.), Duke Molecular Physiology Institute (R.W.M.), Duke Clinical Research Institute (T.A., C.B.P.), and Division of Medical Genetics (D.D.K.), Department of Pediatrics, Duke University Medical Center, Durham, NC.

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http://dx.doi.org/10.1161/CIRCULATIONAHA.114.011647DOI Listing
March 2015

Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE)-like phenotype in a patient with a novel heterozygous POLG mutation.

J Neurol 2014 Sep 15;261(9):1818-9. Epub 2014 Jul 15.

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center 103856, Durham, NC, 27710, USA.

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http://dx.doi.org/10.1007/s00415-014-7428-2DOI Listing
September 2014

Adjunctive albuterol enhances the response to enzyme replacement therapy in late-onset Pompe disease.

FASEB J 2014 May 17;28(5):2171-6. Epub 2014 Jan 17.

1Duke University Medical Center, Box 103856, Durham, NC 27710, USA.

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http://dx.doi.org/10.1096/fj.13-241893DOI Listing
May 2014

The upstream enhancer elements of the G6PC promoter are critical for optimal G6PC expression in murine glycogen storage disease type Ia.

Mol Genet Metab 2013 Nov 25;110(3):275-80. Epub 2013 Jun 25.

Section on Cellular Differentiation, Program on Developmental Endocrinology and Genetics, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD 20892, USA.

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http://dx.doi.org/10.1016/j.ymgme.2013.06.014DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3898731PMC
November 2013

Elevation of guanidinoacetate in newborn dried blood spots and impact of early treatment in GAMT deficiency.

Mol Genet Metab 2013 Jun 16;109(2):215-7. Epub 2013 Mar 16.

Department of Pediatrics, Division of Medical Genetics, Duke University Medical Center, Durham, NC, USA.

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http://dx.doi.org/10.1016/j.ymgme.2013.03.003DOI Listing
June 2013

Adjunctive β2-agonists reverse neuromuscular involvement in murine Pompe disease.

FASEB J 2013 Jan 19;27(1):34-44. Epub 2012 Sep 19.

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, North Carolina 27710, USA.

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http://dx.doi.org/10.1096/fj.12-207472DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3528321PMC
January 2013

Aerobic training as an adjunctive therapy to enzyme replacement in Pompe disease.

Mol Genet Metab 2012 Nov 15;107(3):469-79. Epub 2012 Sep 15.

Department of Pediatrics and Medicine, Neuromuscular Clinic, McMaster University, Hamilton, Ontario, Canada.

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http://dx.doi.org/10.1016/j.ymgme.2012.09.010DOI Listing
November 2012

In search of proof-of-concept: gene therapy for glycogen storage disease type Ia.

Authors:
Dwight D Koeberl

J Inherit Metab Dis 2012 Jul 7;35(4):671-8. Epub 2012 Feb 7.

Division of Medical Genetics, Duke University Medical Center, Durham, NC, USA.

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http://dx.doi.org/10.1007/s10545-012-9454-5DOI Listing
July 2012

Deficiency in MyD88 Signaling Results in Decreased Antibody Responses to an Adeno-Associated Virus Vector in Murine Pompe Disease.

Biores Open Access 2012 Jun;1(3):109-14

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center , Durham, North Carolina.

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http://dx.doi.org/10.1089/biores.2012.0217DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3559236PMC
June 2012

Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe disease.

Hum Gene Ther 2012 May 29;23(5):460-72. Epub 2012 Mar 29.

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

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http://dx.doi.org/10.1089/hum.2011.063DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3360500PMC
May 2012

β2 Agonists enhance the efficacy of simultaneous enzyme replacement therapy in murine Pompe disease.

Mol Genet Metab 2012 Feb 11;105(2):221-7. Epub 2011 Nov 11.

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

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http://dx.doi.org/10.1016/j.ymgme.2011.11.005DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3264842PMC
February 2012

Hepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vector.

Mol Ther 2011 Nov 5;19(11):1961-70. Epub 2011 Jul 5.

Department of Pediatrics, Division of Medical Genetics, Duke University Medical Center, Durham, North Carolina 27710, USA.

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http://dx.doi.org/10.1038/mt.2011.126DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3222521PMC
November 2011

Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle.

Mol Genet Metab 2011 Jun 13;103(2):107-12. Epub 2011 Feb 13.

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

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http://dx.doi.org/10.1016/j.ymgme.2011.02.006DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3101281PMC
June 2011

Metabolic myopathies: clinical features and diagnostic approach.

Rheum Dis Clin North Am 2011 May;37(2):201-17, vi

Division of Pediatric Neurology, Department of Pediatrics, Duke University Medical Center, DUMC Box 3936, Durham, NC 27710, USA.

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http://dx.doi.org/10.1016/j.rdc.2011.01.004DOI Listing
May 2011

The role of hepatocyte hemojuvelin in the regulation of bone morphogenic protein-6 and hepcidin expression in vivo.

J Biol Chem 2010 May 2;285(22):16416-23. Epub 2010 Apr 2.

Department of Cell and Developmental Biology, Oregon Health & Science University, Portland, Oregon 97239, USA.

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http://www.jbc.org/lookup/doi/10.1074/jbc.M110.109488
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http://dx.doi.org/10.1074/jbc.M110.109488DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2878008PMC
May 2010

Hepatocyte-targeted HFE and TFR2 control hepcidin expression in mice.

Blood 2010 Apr 22;115(16):3374-81. Epub 2010 Feb 22.

Department of Cell and Developmental Biology, Oregon Health & Science University, Portland, OR, USA.

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http://www.bloodjournal.org/cgi/doi/10.1182/blood-2009-09-24
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http://dx.doi.org/10.1182/blood-2009-09-245209DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2858491PMC
April 2010

Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease.

Mol Ther 2010 Feb 18;18(2):353-60. Epub 2009 Aug 18.

Department of Pediatrics, Division of Medical Genetics, Duke University Medical Center, Durham, North Carolina, USA.

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http://dx.doi.org/10.1038/mt.2009.195DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2818301PMC
February 2010

Immunomodulatory gene therapy in lysosomal storage disorders.

Curr Gene Ther 2009 Dec;9(6):503-10

Division of Medical Genetics/Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2893380PMC
December 2009

Impaired clearance of accumulated lysosomal glycogen in advanced Pompe disease despite high-level vector-mediated transgene expression.

J Gene Med 2009 Oct;11(10):913-20

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

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http://doi.wiley.com/10.1002/jgm.1372
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http://dx.doi.org/10.1002/jgm.1372DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3622249PMC
October 2009

Liver transplantation for glycogen storage disease type Ia.

J Hepatol 2009 Sep 17;51(3):483-90. Epub 2009 Jun 17.

Department of Surgery, Duke University Medical Center, Durham, NC, USA.

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http://dx.doi.org/10.1016/j.jhep.2009.05.026DOI Listing
September 2009

Activation of glycolysis and apoptosis in glycogen storage disease type Ia.

Mol Genet Metab 2009 Aug 10;97(4):267-71. Epub 2009 Apr 10.

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

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http://dx.doi.org/10.1016/j.ymgme.2009.04.003DOI Listing
August 2009

Gene therapy for inhereted metabolic disorders in companion animals.

ILAR J 2009 ;50(2):122-7

Division of Medical Genetics of the Department of Pediatrics, Duke University Medical Center in Durham, North Carolina 27710, USA.

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http://dx.doi.org/10.1093/ilar.50.2.122DOI Listing
June 2009

Age-related efficacy with an AAV vector in Fabry disease mice.

Authors:
Dwight D Koeberl

Mol Genet Metab 2009 Mar 10;96(3):83-4. Epub 2008 Dec 10.

Department of Pediatrics, Division of Medical Genetics, Duke University Medical Center, DUMC Box 103856, Durham, NC 27710, USA.

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http://dx.doi.org/10.1016/j.ymgme.2008.10.014DOI Listing
March 2009

Improvement with ongoing Enzyme Replacement Therapy in advanced late-onset Pompe disease: a case study.

Mol Genet Metab 2008 Dec 18;95(4):233-5. Epub 2008 Oct 18.

Division of Physical Therapy, Department of Community and Family Medicine, Duke University Medical Center, Durham, North Carolina, NC 27708, USA.

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http://dx.doi.org/10.1016/j.ymgme.2008.09.001DOI Listing
December 2008

A Delphi-based consensus clinical practice protocol for the diagnosis and management of 3-methylcrotonyl CoA carboxylase deficiency.

Mol Genet Metab 2008 Apr 21;93(4):363-70. Epub 2007 Dec 21.

Department of Pediatrics, University of Rochester School of Medicine and Dentistry, 601 Elmwood Avenue, Box 777, Rochester, NY 14642, USA.

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http://dx.doi.org/10.1016/j.ymgme.2007.11.002DOI Listing
April 2008

AAV vector-mediated reversal of hypoglycemia in canine and murine glycogen storage disease type Ia.

Mol Ther 2008 Apr 11;16(4):665-72. Epub 2008 Mar 11.

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, North Carolina 27710, USA.

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http://dx.doi.org/10.1038/mt.2008.15DOI Listing
April 2008

Resection of hepatocellular adenoma in patients with glycogen storage disease type Ia.

J Hepatol 2007 Nov 18;47(5):658-63. Epub 2007 Jun 18.

Department of Surgery, Duke University Medical Center, Box 3247, Durham, NC 27710, USA.

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http://dx.doi.org/10.1016/j.jhep.2007.05.012DOI Listing
November 2007

Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance.

Am J Hum Genet 2007 Nov 21;81(5):1042-9. Epub 2007 Sep 21.

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

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http://dx.doi.org/10.1086/522236DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2265658PMC
November 2007

Efficacy of helper-dependent adenovirus vector-mediated gene therapy in murine glycogen storage disease type Ia.

Mol Ther 2007 Jul 15;15(7):1253-8. Epub 2007 May 15.

Division of Medical Genetics, Duke University Medical Center, Durham, North Carolina, USA.

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http://dx.doi.org/10.1038/sj.mt.6300188DOI Listing
July 2007

Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II.

Mol Ther 2006 Dec 20;14(6):822-30. Epub 2006 Sep 20.

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

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http://dx.doi.org/10.1016/j.ymthe.2006.08.001DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2709843PMC
December 2006

Clinical experience with array CGH: case presentations from nine months of practice.

Am J Med Genet A 2006 Oct;140(19):2050-6

Department of Pediatrics, Division of Clinical Genetics, Duke University Medical Center, Durham, North Carolina 27516, USA.

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http://doi.wiley.com/10.1002/ajmg.a.31417
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http://dx.doi.org/10.1002/ajmg.a.31417DOI Listing
October 2006

Vector-related tumorigenesis not found in ornithine transcarbamylase-deficient mice.

Authors:
Dwight D Koeberl

Mol Ther 2006 Jul 5;14(1):1-2. Epub 2006 Jun 5.

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http://dx.doi.org/10.1016/j.ymthe.2006.05.011DOI Listing
July 2006

Glutaric acidemia type 1 in patients of Lumbee heritage from North Carolina.

Mol Genet Metab 2006 May 8;88(1):90-2. Epub 2006 Feb 8.

Department of Pediatrics, University of North Carolina, Chapel Hill, NC 27599, USA.

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http://dx.doi.org/10.1016/j.ymgme.2005.12.008DOI Listing
May 2006

Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II.

Mol Ther 2005 Nov 6;12(5):876-84. Epub 2005 Jul 6.

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

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http://dx.doi.org/10.1016/j.ymthe.2005.04.024DOI Listing
November 2005

Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter.

Mol Ther 2005 Jun;11(6):889-98

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

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http://dx.doi.org/10.1016/j.ymthe.2005.01.012DOI Listing
June 2005

Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II.

Mol Ther 2005 Jan;11(1):57-65

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

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http://dx.doi.org/10.1016/j.ymthe.2004.10.004DOI Listing
January 2005

Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector.

Mol Ther 2003 Feb;7(2):193-201

Division of Medical Genetics, Duke University Medical Center, Durham, NC 27710, USA.

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http://dx.doi.org/10.1016/s1525-0016(02)00055-2DOI Listing
February 2003

Flupirtine blocks apoptosis in batten patient lymphoblasts and in human postmitotic CLN3- and CLN2-deficient neurons.

Ann Neurol 2002 Apr;51(4):448-66

Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

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April 2002