K. Ponder

发表

Evaluation of relative promoter strength in primary hepatocytes using optimized lipofection.

K. Ponder, R. Dunbar, D. R. Wilson, 1991, Human gene therapy.

Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and management

W. Roberts, R. Giugliani, K. Ponder, 2011, Journal of Inherited Metabolic Disease.

Analysis of liver development, regeneration, and carcinogenesis by genetic marking studies

K. Ponder, 1996, FASEB journal : official publication of the Federation of American Societies for Experimental Biology.

Ras-transduced diethylnitrosamine-treated hepatocytes develop into cancers of mixed phenotype in vivo.

M. W. Flye, K. Ponder, E. Brunt, 1995, Cancer research.

The effect of neonatal gene therapy on skeletal manifestations in mucopolysaccharidosis VII dogs after a decade.

M. Haskins, K. Ponder, M. Casal, 2013, Molecular genetics and metabolism.

Neonatal intravenous injection of a gammaretroviral vector has a low incidence of tumor induction in mice.

K. Ponder, Xiucui Ma, Mindy Tittiger, 2008, Human gene therapy.

Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation.

K. Ponder, Baomei Wang, A. Hennig, 2007, Molecular therapy : the journal of the American Society of Gene Therapy.

Gene therapy for mucopolysaccharidosis

M. Haskins, K. Ponder, 2007, Expert opinion on biological therapy.

Immunology of neonatal gene transfer.

K. Ponder, 2007, Current gene therapy.

Gene therapy for hemophilia

K. Ponder, 2006, Current opinion in hematology.

Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B.

S. Cai, Cuihua Gao, D. Bellinger, 2003, Blood.

Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs.

Cuihua Gao, T. O'Malley, M. Haskins, 2002, Molecular therapy : the journal of the American Society of Gene Therapy.

Gene Therapy for Hepatic and Vascular Disorders

M. Hicks, K. Ponder, J. Duncan, 1997 .

Embolization of portal vein branches induces hepatocyte replication in swine: a potential step in hepatic gene therapy.

M. Hicks, S. Cai, K. Ponder, 1999, Radiology.

Systemic gene therapy for cardiovascular disease.

K. Ponder, 1999, Trends in cardiovascular medicine.

High expression reduces an antibody response after neonatal gene therapy with B domain‐deleted human factor VIII in mice

K. Ponder, X. Ma, M. Mei, 2007, Journal of thrombosis and haemostasis : JTH.

Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII

C. Vite, James M. Wilson, M. Haskins, 2015, Molecular therapy : the journal of the American Society of Gene Therapy.

Gene transfer to the liver

K. Ponder, 2005 .

Intramuscular injection of an adenoviral vector expressing hepatocyte growth factor facilitates hepatic transduction with a retroviral vector in mice.

S. Cai, K. Ponder, S. Kennedy, 1999, Human gene therapy.

Therapeutic levels of human protein C in rats after retroviral vector-mediated hepatic gene therapy.

S. Cai, M. W. Flye, K. Ponder, 1998, The Journal of clinical investigation.

Portal branch occlusion safely facilitates in vivo retroviral vector transduction of rat liver.

S. Cai, M. W. Flye, K. Ponder, 1996, Human gene therapy.

Gene Delivery for Systemic Expression: Plasmid, Retroviral, and Adenoviral Approaches

K. Ponder, 1997 .

Status of therapeutic gene transfer to treat cardiovascular disease in dogs and cats.

H. Sweeney, M. Haskins, K. Ponder, 2011, Journal of veterinary cardiology : the official journal of the European Society of Veterinary Cardiology.

Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs.

M. Haskins, M. Sands, K. Ponder, 2004, Molecular genetics and metabolism.

855. Stable Transduction of Hepatocytes Following Intravenous Retroviral Gene Therapy in Seven-Week-Old Dogs with Mucopolysaccharidosis (MPS) VII|[ast]|

G. Aguirre, T. O'Malley, M. Haskins, 2004 .

Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy.

M. Haskins, M. Sands, K. Ponder, 2002, Molecular therapy : the journal of the American Society of Gene Therapy.

Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.

L. Naldini, R. Brambilla, A. Biffi, 2010, Blood.

Genetic Control of Human Alpha-1-Antitrypsin and Hepatic Gene Therapy

K. Ponder, S. Woo, R. Sifers, 1992 .

Quantitative evaluation of liver-specific promoters from retroviral vectors after in vivo transduction of hepatocytes.

M. W. Flye, K. Ponder, D. Hafenrichter, 1994, Blood.

Liver-directed gene therapy: quantitative evaluation of promoter elements by using in vivo retroviral transduction.

M. W. Flye, K. Ponder, D. Hafenrichter, 1994, Proceedings of the National Academy of Sciences of the United States of America.

Pathogenesis of mitral valve disease in mucopolysaccharidosis VII dogs.

R. Giugliani, M. Haskins, K. Ponder, 2013, Molecular genetics and metabolism.

Lipopolysaccharide results in a marked decrease in hepatocyte nuclear factor 4α in rat liver

F. Sladek, S. Cai, Cuihua Gao, 2001 .

Article Title:

Cuihua Gao, D. Bellinger, M. Sands, 2003 .

Human gene therapy: Present and future

M. Kay, K. Ponder, S. Woo, 1992, Breast Cancer Research and Treatment.

Lovastatin decreases mortality and improves liver functions in fulminant hepatic failure from 90% partial hepatectomy in rats.

S. Cai, M. W. Flye, K. Ponder, 2000, Journal of hepatology.

So why is the leukemogenic mechanism in humans so indirect ?

K. Ponder, 2008 .

Gene therapy goes to the dogs

K. Ponder, 2006 .

Gene therapy goes to the dogs combined

K. Ponder, 2006 .

In vivo transduction of hematopoietic stem cells after neonatal intravenous injection of an amphotropic retroviral vector in mice.

T. O'Malley, M. Haskins, M. Sands, 2004, Molecular therapy : the journal of the American Society of Gene Therapy.

Expression of human alpha 1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes.

M. Kay, T. Liu, M. Finegold, 1992, Proceedings of the National Academy of Sciences of the United States of America.

Upregulation of elastase activity in aorta in mucopolysaccharidosis I and VII dogs may be due to increased cytokine expression.

M. Haskins, K. Ponder, M. Whyte, 2010, Molecular genetics and metabolism.

Mechanism of shortened bones in mucopolysaccharidosis VII.

K. Ponder, J. Metcalf, Fanxin Long, 2009, Molecular genetics and metabolism.

Effect of neonatal gene therapy on lumbar spine disease in mucopolysaccharidosis VII dogs.

D. Elliott, John T. Martin, Lachlan J. Smith, 2012, Molecular genetics and metabolism.

Expression in blood cells may contribute to biochemical and pathological improvements after neonatal intravenous gene therapy for mucopolysaccharidosis VII in dogs.

C. Vite, T. O'Malley, M. Haskins, 2006, Molecular genetics and metabolism.

Pathogenesis of aortic dilatation in mucopolysaccharidosis VII mice may involve complement activation.

R. Knutsen, R. Mecham, J. Atkinson, 2011, Molecular genetics and metabolism.

Therapeutic levels of functional human factor X in rats after retroviral-mediated hepatic gene therapy.

S. Cai, M. W. Flye, T. Okuyama, 1997, Blood.

Retroviral vector sequences may interact with some internal promoters and influence expression.

K. Ponder, J. Holschen, S. Kennedy, 1996, Human gene therapy.

C/EBPbeta contributes to hepatocyte growth factor-induced replication of rodent hepatocytes.

Cuihua Gao, K. Ponder, Bin Wang, 2005, Journal of hepatology.

Immune response hinders therapy for lysosomal storage diseases.

K. Ponder, 2008, The Journal of clinical investigation.

Lipopolysaccharide results in a marked decrease in hepatocyte nuclear factor 4 alpha in rat liver.

F. Sladek, S. Cai, K. Ponder, 2001, Hepatology.

Liver-directed gene therapy: evaluation of liver specific promoter elements.

M. W. Flye, K. Ponder, D. Hafenrichter, 1994, The Journal of surgical research.

Improved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I mice

K. Ponder, K. Ohlemiller, A. Kovács, 2008, The journal of gene medicine.

Immune response after neonatal transfer of a human factor IX-expressing retroviral vector in dogs, cats, and mice.

M. Haskins, D. Bellinger, T. Nichols, 2007, Thrombosis research.

Delivery of a retroviral vector expressing human beta-glucuronidase to the liver and spleen decreases lysosomal storage in mucopolysaccharidosis VII mice.

M. Haskins, M. Sands, K. Ponder, 2000, Molecular therapy : the journal of the American Society of Gene Therapy.

CHAPTER 4 Vectors of Gene Therapy

K. Ponder, 2022 .

Merry christmas for patients with hemophilia B.

K. Ponder, 2011, The New England journal of medicine.

More on: are randomized clinical trials the only truth? Not always

P. Mannucci, C. Kessler, P. Monahan, 2006, Journal of thrombosis and haemostasis : JTH.

Unresolved issues in diagnosis and management of inherited bleeding disorders in the perinatal period: A White Paper of the Perinatal Task Force of the Medical and Scientific Advisory Council of the National Hemophilia Foundation, USA

K. Ponder, J. Soucie, W. Hoots, 2006, Haemophilia : the official journal of the World Federation of Hemophilia.

Should gene therapy be used for newborns with hemophilia?

K. Ponder, 2003, Molecular therapy : the journal of the American Society of Gene Therapy.

Hemophilia gene therapy: a Holy Grail found.

K. Ponder, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

Absence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy

R. Sarkar, D. Bellinger, T. Nichols, 2005, Proceedings of the National Academy of Sciences of the United States of America.

Walk a mile in the moccasins of people with haemophilia

K. Ponder, A. Srivastava, 2008, Haemophilia : the official journal of the World Federation of Hemophilia.

Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I.

C. Vite, M. Mcentee, M. Haskins, 2010, Molecular genetics and metabolism.

Correction of clinical manifestations of canine mucopolysaccharidosis I with neonatal retroviral vector gene therapy.

C. Vite, G. Aguirre, M. Haskins, 2007, Molecular therapy : the journal of the American Society of Gene Therapy.

Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

G. Aguirre, T. O'Malley, M. Haskins, 2002, Proceedings of the National Academy of Sciences of the United States of America.

Effects of Neonatal Enzyme Replacement Therapy and Simvastatin Treatment on Cervical Spine Disease in Mucopolysaccharidosis I Dogs

N. Malhotra, J. Chiaro, Lachlan J. Smith, 2014, Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research.

The effect of Tlr4 and/or C3 deficiency and of neonatal gene therapy on skeletal disease in mucopolysaccharidosis VII mice.

K. Ponder, Susan Wu, Elizabeth M Xing, 2015, Molecular genetics and metabolism.

The effect of neonatal gene therapy with a gamma retroviral vector on cardiac valve disease in mucopolysaccharidosis VII dogs after a decade.

M. Haskins, K. Ponder, M. Casal, 2013, Molecular genetics and metabolism.

Neonatal gene therapy with a gamma retroviral vector in mucopolysaccharidosis VI cats.

D. Brooks, T. O'Malley, M. Haskins, 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

A review of gene therapy in canine and feline models of lysosomal storage disorders.

C. Vite, M. Haskins, K. Ponder, 2015, Human gene therapy. Clinical development.

A review of gene therapy in canine and feline models of lysosomal storage disorders.

C. Vite, M. Haskins, K. Ponder, 2015, Human gene therapy. Clinical development.

Mouse hepatocytes migrate to liver parenchyma and function indefinitely after intrasplenic transplantation.

F D Ledley, J. Chowdhury, F. Ledley, 1991, Proceedings of the National Academy of Sciences of the United States of America.

Experiments in transgenic mice show that hepatocytes are the source for postnatal liver growth and do not stream

K. Ponder, S. Kennedy, S. Rettinger, 1995, Hepatology.

Gene Therapy for Primary and Metastatic Cancer to the Liver

K. Ponder, M. Flye, 2000 .

PROMOTERS CAN INTERACT WITH RETROVIRAL SEQUENCES 161

K. Ponder, J. Holschen, Wu Xiaoyun, 2008 .

Liver-directed gene therapy: a retroviral vector with a complete LTR and the ApoE enhancer-alpha 1-antitrypsin promoter dramatically increases expression of human alpha 1-antitrypsin in vivo.

M. W. Flye, T. Okuyama, K. Ponder, 1996, Human gene therapy.

Cutting Edge: Treatment of Complement Regulatory Protein Deficiency by Retroviral In Vivo Gene Therapy1

J. Atkinson, K. Ponder, D. Spitzer, 2006, The Journal of Immunology.

In Vivo Gene Therapy Regulatory Protein Deficiency by Retroviral Cutting Edge: Treatment of Complement

J. Atkinson, K. Ponder, D. Spitzer, 2006 .

In vivo hepatocyte transduction with retrovirus during in-flow occlusion.

M. W. Flye, K. Ponder, D. Hafenrichter, 1993, The Journal of surgical research.

Upregulation of elastase proteins results in aortic dilatation in mucopolysaccharidosis I mice.

R. Knutsen, R. Mecham, K. Ponder, 2008, Molecular genetics and metabolism.

Lipopolysaccharide potentiates the effect of hepatocyte growth factor upon replication in lung, thyroid, spleen, and colon in rats in vivo.

K. Ponder, S. Kennedy, C. Gao, 2001, Molecular therapy : the journal of the American Society of Gene Therapy.

Lipopolysaccharide potentiates the effect of hepatocyte growth factor on hepatocyte replication in rats by augmenting AP‐1 activity

S. Cai, M. W. Flye, Cuihua Gao, 1999, Hepatology.

A self-inactivating gamma-retroviral vector reduces manifestations of mucopolysaccharidosis I in mice.

A. Schambach, K. Ponder, K. Ohlemiller, 2010, Molecular therapy : the journal of the American Society of Gene Therapy.

Radiographic evaluation of bones and joints in mucopolysaccharidosis I and VII dogs after neonatal gene therapy.

M. Haskins, K. Ponder, R. Herati, 2008, Molecular genetics and metabolism.

Mucopolysaccharidosis I cats mount a cytotoxic T lymphocyte response after neonatal gene therapy that can be blocked with CTLA4-Ig.

Bin Wang, M. Haskins, K. Ponder, 2006, Molecular therapy : the journal of the American Society of Gene Therapy.

Altered lumbar spine structure, biochemistry, and biomechanical properties in a canine model of mucopolysaccharidosis type VII

Spencer E. Szczesny, D. Elliott, John T. Martin, 2010, Journal of orthopaedic research : official publication of the Orthopaedic Research Society.

Pathogenesis of lumbar spine disease in mucopolysaccharidosis VII.

D. Elliott, Lachlan J. Smith, R. Giugliani, 2012, Molecular genetics and metabolism.

Postnatal progression of bone disease in the cervical spines of mucopolysaccharidosis I dogs.

D. Elliott, J. Chiaro, Lachlan J. Smith, 2013, Bone.

A Self-inactivating γ-Retroviral Vector Reduces Manifestations of Mucopolysaccharidosis I in Mice.

A. Schambach, K. Ponder, K. Ohlemiller, 2010, Molecular therapy : the journal of the American Society of Gene Therapy.

Effect of neonatal administration of a retroviral vector expressing alpha-L-iduronidase upon lysosomal storage in brain and other organs in mucopolysaccharidosis I mice.

K. Ponder, Xiucui Ma, Yuli Liu, 2007, Molecular genetics and metabolism.

Clinical characterization of cardiovascular abnormalities associated with feline mucopolysaccharidosis I and VI

M. Haskins, K. Ponder, F. Shofer, 2008, Journal of Inherited Metabolic Disease.

Gene Therapy Ameliorates Cardiovascular Disease in Dogs With Mucopolysaccharidosis VII

T. O'Malley, M. Haskins, K. Ponder, 2004, Circulation.

Retroviral-vector-mediated gene therapy to mucopolysaccharidosis I mice improves sensorimotor impairments and other behavioral deficits

D. Wozniak, R. Giugliani, K. Ponder, 2013, Journal of Inherited Metabolic Disease.

Evidence of a progressive motor dysfunction in Mucopolysaccharidosis type I mice

R. Giugliani, U. Matte, K. Ponder, 2012, Behavioural Brain Research.

Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases

J. Joung, C. Barbas, A. Bogdanove, 2013, Molecular therapy : the journal of the American Society of Gene Therapy.

Gene therapy for ocular problems in mucopolysaccharidosis: an experimental and promising approach with benefits in animal models – a review

K. Ponder, A. Auricchio, 2010 .

Alterations in enzymatic functions in hepatocytes and hepatocellular carcinomas from Ras‐transduced livers resemble the effects of insulin

K. Ponder, E. Brunt, Y. Lin, 1996, Hepatology.

FIXing factor VIII inhibitors.

K. Ponder, 2012, Blood.

Gene therapy for mucopolysaccharidosis VII: evaluation of intrathecal rAAV vectors in the canine model

C. Vite, James M. Wilson, M. Haskins, 2014 .

Vectors in Gene Therapy

K. Ponder, 2002 .

A more clinically relevant mouse model of hemophilia B

K. Ponder, 2004 .

Gene therapy for hemophilia

K. Ponder, R. Schwaab, T. Albert, 2002, Current opinion in hematology.

CMV-beta-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1 alpha promoter and results in therapeutic levels of human factor X in mice.

B. Byrne, T. Flotte, M. Sands, 2001, Human gene therapy.

Postnatal progression of bone disease in the cervical spines of mucopolysaccharidosis I dogs.

D. Elliott, J. Chiaro, Lachlan J. Smith, 2013, Bone.

A Self-inactivating γ-Retroviral Vector Reduces Manifestations of Mucopolysaccharidosis I in Mice.

A. Schambach, K. Ponder, K. Ohlemiller, 2010, Molecular therapy : the journal of the American Society of Gene Therapy.