Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency

Conditional disruption of hepatic carbamoyl phosphate synthetase 1 in mice results in hyperammonemia without orotic aciduria and can be corrected by liver-directed gene therapy

(2018) Suhail Khoja et al.   MOLECULAR GENETICS AND METABOLISM

Human hepatocyte transplantation corrects the inherited metabolic liver disorder arginase deficiency in mice

(2018) Stephanie A.K. Angarita et al.   MOLECULAR GENETICS AND METABOLISM

“Cerebral Palsy” in a Patient With Arginase Deficiency

(2018) Amanda Jichlinski et al.   Seminars in Pediatric Neurology

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

(2018) Ding An et al.   Cell Reports

Prenatal gene therapy offers the earliest possible cure

(2018) Sarah DeWeerdt   NATURE

Analysis of Liver Offers to Pediatric Candidates on the Transplant Wait List

(2017) Evelyn K. Hsu et al.   GASTROENTEROLOGY

Newborn screening for hyperargininemia due to arginase 1 deficiency

(2017) Bradford L. Therrell et al.   MOLECULAR GENETICS AND METABOLISM

Systemic delivery of factor IX messenger RNA for protein replacement therapy

(2017) Suvasini Ramaswamy et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

(2017) Ding An et al.   Cell Reports

Lipid nanoparticles for targeted siRNA delivery – going from bench to bedside

(2016) Victor Kotelianski et al.   International Journal of Nanomedicine

Rescue of the Functional Alterations of Motor Cortical Circuits in Arginase Deficiency by Neonatal Gene Therapy

(2016) Gloria Cantero et al.   JOURNAL OF NEUROSCIENCE

Human recombinant arginase enzyme reduces plasma arginine in mouse models of arginase deficiency

(2015) Lindsay C. Burrage et al.   HUMAN MOLECULAR GENETICS

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

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

Treatment of arginase deficiency revisited: guanidinoacetate as a therapeutic target and biomarker for therapeutic monitoring

(2014) Wajdi Amayreh et al.   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Minimal ureagenesis is necessary for survival in the murine model of hyperargininemia treated by AAV-based gene therapy

(2014) C Hu et al.   GENE THERAPY

Myocyte-mediated Arginase Expression Controls Hyperargininemia but not Hyperammonemia in Arginase-deficient Mice

(2014) Chuhong Hu et al.   MOLECULAR THERAPY

AAV-based gene therapy prevents neuropathology and results in normal cognitive development in the hyperargininemic mouse

(2013) E K Lee et al.   GENE THERAPY

Lethal phenotype in conditional late-onset arginase 1 deficiency in the mouse

(2013) Jennifer Kasten et al.   MOLECULAR GENETICS AND METABOLISM

The incidence of urea cycle disorders

(2013) Marshall L. Summar et al.   MOLECULAR GENETICS AND METABOLISM

AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in mice

(2012) C Hu et al.   GENE THERAPY

Long-term Survival of the Juvenile Lethal Arginase-deficient Mouse With AAV Gene Therapy

(2012) Eun K. Lee et al.   MOLECULAR THERAPY

Clinical Features and Neurologic Progression of Hyperargininemia

(2012) Daniel R. Carvalho et al.   PEDIATRIC NEUROLOGY

Expression of therapeutic proteins after delivery of chemically modified mRNA in mice

(2011) Michael S D Kormann et al.   NATURE BIOTECHNOLOGY

RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy

(2010) Chuhong Hu et al.   JOURNAL OF GENE MEDICINE

Guanidino compound levels in blood, cerebrospinal fluid, and post-mortem brain material of patients with argininemia

(2010) Joshua L. Deignan et al.   MOLECULAR GENETICS AND METABOLISM

Short-term Correction of Arginase Deficiency in a Neonatal Murine Model With a Helper-dependent Adenoviral Vector

(2009) Chia-Ling Gau et al.   MOLECULAR THERAPY

Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors

(2008) Sharon C Cunningham et al.   MOLECULAR THERAPY

Increased plasma and tissue guanidino compounds in a mouse model of hyperargininemia

(2007) Joshua L. Deignan et al.   MOLECULAR GENETICS AND METABOLISM