ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia

Enhancement of liver-directed transgene expression at initial and repeat doses of AAV vectors admixed with ImmTOR nanoparticles

(2021) Petr O. Ilyinskii et al.   Science Advances

Impaired mitophagy links mitochondrial disease to epithelial stress in methylmalonyl-CoA mutase deficiency

(2020) Alessandro Luciani et al.   Nature Communications

Capsid-specific removal of circulating antibodies to adeno-associated virus vectors

(2020) Berangere Bertin et al.   Scientific Reports

Successful Transduction with AAV Vectors after Selective Depletion of Anti-AAV Antibodies by Immunoadsorption

(2020) Alejandro Orlowski et al.   Molecular Therapy-Methods & Clinical Development

Management of Neuroinflammatory Responses to AAV-Mediated Gene Therapies for Neurodegenerative Diseases

(2020) Barbara A. Perez et al.   Brain Sciences

IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies

(2020) Christian Leborgne et al.   NATURE MEDICINE

Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B

(2020) Lindsey A. George et al.   MOLECULAR THERAPY

Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients

(2020) Xiaoxia Shi et al.   Molecular Therapy-Methods & Clinical Development

Optimization of Dexamethasone Administration for Maintaining Global Transduction Efficacy of Adeno-Associated Virus Serotype 9

(2019) Zheng Chai et al.   HUMAN GENE THERAPY

Therapeutic hFIX activity achieved after single AAV5-hFIX treatment in hemophilia B patients and NHPs with pre-existing anti-AAV5 NABs

(2019) Anna Majowicz et al.   Molecular Therapy-Methods & Clinical Development

Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future

(2019) Randy Joseph Chandler et al.   HUMAN GENE THERAPY

Gene Therapy

(2019) Katherine A. High et al.   NEW ENGLAND JOURNAL OF MEDICINE

Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

(2019) Allison M. Keeler et al.   Annual Review of Virology

Long-term efficacy and safety of mRNA therapy in two murine models of methylmalonic acidemia

(2019) Ding An et al.   EBioMedicine

Prevalence of Anti-Adeno-Associated Virus Immune Responses in International Cohorts of Healthy Donors

(2019) Anita Kruzik et al.   Molecular Therapy-Methods & Clinical Development

Tolerogenic nanoparticles restore the antitumor activity of recombinant immunotoxins by mitigating immunogenicity

(2018) Ronit Mazor et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Liver Transplantation for Propionic Acidemia and Methylmalonic Acidemia: Perioperative Management and Clinical Outcomes

(2018) Kristen Critelli et al.   LIVER TRANSPLANTATION

Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration

(2018) Amine Meliani et al.   Nature Communications

Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectors

(2017) Marianna Hösel et al.   HEPATOLOGY

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

(2017) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

(2017) Ding An et al.   Cell Reports

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape

(2017) Federico Mingozzi et al.   Annual Review of Virology

Tolerogenic nanoparticles to induce immunologic tolerance: Prevention and reversal of FVIII inhibitor formation

(2016) Ai-Hong Zhang et al.   CELLULAR IMMUNOLOGY

Methylmalonic and propionic acidemias

(2016) Jamie L. Fraser et al.   CURRENT OPINION IN PEDIATRICS

Improving the efficacy and safety of biologic drugs with tolerogenic nanoparticles

(2016) Takashi K. Kishimoto et al.   Nature Nanotechnology

Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy

(2015) Randy J. Chandler et al.   JOURNAL OF CLINICAL INVESTIGATION

The phenotypic spectrum of organic acidurias and urea cycle disorders. Part 2: the evolving clinical phenotype

(2015) Stefan Kölker et al.   JOURNAL OF INHERITED METABOLIC DISEASE

The phenotypic spectrum of organic acidurias and urea cycle disorders. Part 1: the initial presentation

(2015) Stefan Kölker et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Treatment of Methylmalonic Acidemia by Liver or Combined Liver-Kidney Transplantation

(2015) Anna-Kaisa Niemi et al.   JOURNAL OF PEDIATRICS

In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector

(2015) Eric Zinn et al.   Cell Reports

Proposed guidelines for the diagnosis and management of methylmalonic and propionic acidemia

(2014) Matthias R Baumgartner et al.   Orphanet Journal of Rare Diseases

Polymeric synthetic nanoparticles for the induction of antigen-specific immunological tolerance

(2014) Roberto A. Maldonado et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Plasmapheresis Eliminates the Negative Impact of AAV Antibodies on Microdystrophin Gene Expression Following Vascular Delivery

(2013) LG Chicoine et al.   MOLECULAR THERAPY

Targeting proximal tubule mitochondrial dysfunction attenuates the renal disease of methylmalonic acidemia

(2013) I. Manoli et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

(2013) F. Mingozzi et al.   Science Translational Medicine

Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B

(2012) Federico Mingozzi et al.   MOLECULAR THERAPY

Neurocognitive Phenotype of Isolated Methylmalonic Acidemia

(2012) C. J. O'Shea et al.   PEDIATRICS

Adeno-associated virus serotype 9-mediated pulmonary transgene expression: effect of mouse strain, animal gender and lung inflammation

(2011) C Pfeifer et al.   GENE THERAPY

Gene therapy in a murine model of methylmalonic acidemia using rAAV9-mediated gene delivery

(2011) J S Sénac et al.   GENE THERAPY

A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8

(2011) Virginie Monteilhet et al.   MOLECULAR THERAPY

Preoperative Dialysis for Liver Transplantation in Methylmalonic Acidemia

(2011) Koichi Kamei et al.   THERAPEUTIC APHERESIS AND DIALYSIS

Liver-Directed Recombinant Adeno-Associated Viral Gene Delivery Rescues a Lethal Mouse Model of Methylmalonic Acidemia and Provides Long-Term Phenotypic Correction

(2010) Nuria Carrillo-Carrasco et al.   HUMAN GENE THERAPY

AAV2/8-mediated Correction of OTC Deficiency Is Robust in Adult but Not Neonatal Spfash Mice

(2009) Sharon C Cunningham et al.   MOLECULAR THERAPY

Stabilization of blood methylmalonic acid level in methylmalonic acidemia after liver transplantation

(2009) P. W. Chen et al.   PEDIATRIC TRANSPLANTATION

Mitochondrial dysfunction in mut methylmalonic acidemia

(2008) Randy J. Chandler et al.   FASEB JOURNAL

Adenovirus-Mediated Gene Delivery Rescues a Neonatal Lethal Murine Model of mut0 Methylmalonic Acidemia

(2007) Randy J. Chandler et al.   HUMAN GENE THERAPY