Physician

Pediatrics

Dr. Jon A. Wolff is an expert in the field of Pediatrics. He attended Johns Hopkins University School of Medicine. Johns Hopkins University School of Medicine is a one of the best medicals schools. Johns Hopkins University School of Medicine is ranked 3/29 in Research/PrimaryCare. Doctor Jon A. Wolff, MD is also a published doctor. He has 100 paper published. The most recent paper is: Renal transplantation in a patient with methylmalonic acidaemia.

Publications

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• Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD.
• Dose Response in Rodents and Nonhuman Primates After Hydrodynamic Limb Vein Delivery of Naked Plasmid DNA.
• Muscle damage after delivery of naked plasmid DNA into skeletal muscles is batch dependent.
• Systemic gene transfer to skeletal muscle using reengineered AAV vectors.
• Hybrid PET/CT for noninvasive pharmacokinetic evaluation of dynamic PolyConjugates, a synthetic siRNA delivery system.
• Evaluation of hydrodynamic limb vein injections in nonhuman primates.
• Use of Evans blue dye to compare limb muscles in exercised young and old mdx mice.
• Functional efficacy of dystrophin expression from plasmids delivered to mdx mice by hydrodynamic limb vein injection.
• Effect of tissue-specific promoters and microRNA recognition elements on stability of transgene expression after hydrodynamic naked plasmid DNA delivery.
• Improved nutritional management of phenylketonuria by using a diet containing glycomacropeptide compared with amino acids.
• The role of molecular testing and enzyme analysis in the management of hypomorphic citrullinemia.
• Correction of murine PKU following AAV-mediated intramuscular expression of a complete phenylalanine hydroxylating system.
• A single acyl-CoA dehydrogenase is required for catabolism of isoleucine, valine and short-chain fatty acids in Aspergillus nidulans.
• Breaking the bonds: non-viral vectors become chemically dynamic.
• Medium chain acyl-coenzyme A dehydrogenase deficiency in a neonate.
• Dynamic PolyConjugates for targeted in vivo delivery of siRNA to hepatocytes.
• In vivo selection and validation of liver-specific ligands using a new T7 phage peptide display system.
• Magnetic resonance imaging-monitored plasmid DNA delivery in primate limb muscle.
• Progress toward a nonviral gene therapy protocol for the treatment of anemia.
• 2-methylbutyryl-CoA dehydrogenase deficiency in Hmong infants identified by expanded newborn screen.
• Condensation of nonstochiometric DNA/polycation complexes by divalent cations.
• Hepatocyte targeting of nucleic acid complexes and liposomes by a T7 phage p17 peptide.
• Genetic immunization for antibody generation in research animals by intravenous delivery of plasmid DNA.
• Membrane activity and transfection ability of amphipathic polycations as a function of alkyl group size.
• Delivery of siRNA and siRNA expression constructs to adult mammals by hydrodynamic intravascular injection.
• The mechanism of naked DNA uptake and expression.
• A facile nonviral method for delivering genes and siRNAs to skeletal muscle of mammalian limbs.
• Intraarterial delivery of naked plasmid DNA expressing full-length mouse dystrophin in the mdx mouse model of duchenne muscular dystrophy.
• Naked DNA gene transfer in mammalian cells.
• A new peptide ligand that targets particles and heterologous proteins to hepatocytes in vivo.
• Efficient in vitro and in vivo expression of covalently modified plasmid DNA.
• Prospective diagnosis of 2-methylbutyryl-CoA dehydrogenase deficiency in the Hmong population by newborn screening using tandem mass spectrometry.
• The role of a microscopic colloidally stabilized phase in solubilizing oligoamine-condensed DNA complexes.
• Endosomolysis by masking of a membrane-active agent (EMMA) for cytoplasmic release of macromolecules.
• Efficient delivery of siRNA for inhibition of gene expression in postnatal mice.
• The "grand" problem of synthetic delivery.
• The effect of cell division on the cellular dynamics of microinjected DNA and dextran.
• L-carnitine administration reduces number of episodes in cyclic vomiting syndrome.
• Entrapment and condensation of DNA in neutral reverse micelles.
• Surgical procedures for intravascular delivery of plasmid DNA to organs.
• Gene expression from transcriptionally disabled retroviral vectors.
• Alanine decreases the protein requirements of infants with inborn errors of amino acid metabolism.
• Correlation of cognitive, neurologic, and ovarian outcome with the Q188R mutation of the galactose-1-phosphate uridyltransferase gene.
• Cognitive functioning, neurologic status and brain imaging in classical galactosemia.
• Up-regulation of type 1 plasminogen activator inhibitor messenger RNA with thrombotic changes in renal grafts.
• Sickle cell anemia and iron deficiency. Resemblance to sickle thalassemia.
• Intravenously administered lecithin liposomes: a synthetic antiatherogenic lipid particle.
• Grafting genetically modified cells into the rat brain: characteristics of E. coli beta-galactosidase as a reporter gene.
• Neonatal screening for glucose-6-phosphate dehydrogenase deficiency.
• A novel 3'' extension technique using random primers in RNA-PCR.
• Grafting genetically modified cells to the damaged brain: restorative effects of NGF expression.
• The treatment of orbital rhabdomyosarcoma with irradiation and chemotherapy.
• Abnormal somatosensory evoked potentials in patients with classic galactosemia: correlation with neurologic outcome.
• An analysis of the resident match.
• Grafting genetically modified cells to the brain: possibilities for the future.
• Galactosemia: evaluation with MR imaging.
• Progressive renal insufficiency in methylmalonic acidemia.
• Defects of neutrophil chemotaxis and random migration in thalassemia major.
• A longitudinal study of cognitive functioning in patients with classical galactosaemia, including a cohort treated with oral uridine.
• Expression of retrovirally transduced genes in primary cultures of adult rat hepatocytes.
• Metabolism of 1-13C-propionate in vivo in patients with disorders of propionate metabolism.
• Factors affecting long-term stability of Moloney murine leukemia virus-based vectors.
• Co-transplantation of plasmid-transfected myoblasts and myotubes into rat brains enables high levels of gene expression long-term.
• Grafting fibroblasts genetically modified to produce L-dopa in a rat model of Parkinson disease.
• Gene therapy--a primer.
• Rapidly reversible hydrophobization: an approach to high first-pass drug extraction.
• Adult mammalian hepatocyte as target cell for retroviral gene transfer: a model for gene therapy.
• Partial titin cDNA sequence isolated from rabbit cardiac muscle RNA.
• Peripheral nerve in type III glycogenosis: selective involvement of unmyelinated fiber Schwann cells.
• Carnitine reduces fasting ketogenesis in patients with disorders of propionate metabolism.
• Gene transfer and expression in rat cardiac transplants.
• The mouse mutation sarcosinemia (sar) maps to chromosome 2 in a region homologous to human 9q33-q34.
• Postnatal gene transfer into the central nervous system.
• Long-term correction of rat model of Parkinson''s disease by gene therapy.
• Self-amplifying expression from the T7 promoter in 3T3 mouse fibroblasts.
• Characterization of a partial cDNA clone encoding porcine skeletal muscle titin: comparison with rabbit and mouse skeletal muscle titin sequences.
• Novel human pathological mutations. Gene symbol: ASS1. Disease: Citrullinaemia.
• Direct gene transfer into muscle.
• cDNA sequence of rabbit cardiac titin/connectin.
• Long-term survival of autologous muscle grafts in rat brain.
• Renal transplantation in a patient with methylmalonic acidaemia.
• Particle bombardment-mediated gene transfer and expression in rat brain tissues.
• High-efficiency protein synthesis from T7 RNA polymerase transcripts in 3T3 fibroblasts.
• Efficient and sustained transgene expression in mature rat oligodendrocytes in primary culture.
• Functional methionine synthase deficiency due to cblG disorder: a report of two patients and a review.
• Pharmacological enhancement of in vivo foreign gene expression in muscle.
• Intracerebral transplants of primary muscle cells: a potential ''platform'' for transgene expression in the brain.
• sar: a genetic mouse model for human sarcosinemia generated by ethylnitrosourea mutagenesis.
• Quantitative assessment of DNA condensation.
• Primary structure of the kinase domain region of rabbit skeletal and cardiac muscle titin.
• Expression of phenylalanine hydroxylase (PAH) in erythrogenic bone marrow does not correct hyperphenylalaninemia in Pah(enu2) mice.
• An early history of gene transfer and therapy.
• The frequency of revertants in mdx mouse genetic models for Duchenne muscular dystrophy.
• Nonnuclear DNA binding proteins in striated muscle.
• Clinical and biochemical variation and family studies in the multiple acyl-CoA dehydrogenation disorders.
• Transcriptional and phenotypic comparisons of Ppara knockout and siRNA knockdown mice.
• Systemic siRNA delivery via hydrodynamic intravascular injection.
• Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controll...
• Naked DNA transport and expression in mammalian cells.

Schools

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• Harvard Medical School

• Johns Hopkins University School of Medicine

Doctors Specialties

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• Clinical Biochemical Genetics
• Clinical Genetics
• Clinical Molecular Genetics
• Pediatrics

Accepted Insurances

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Education

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• University of California San Diego
• Johns Hopkins University School of Medicine

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