Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease
Published 2021 View Full Article
- Home
- Publications
- Publication Search
- Publication Details
Title
Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease
Authors
Keywords
Krabbe disease, gene therapy, adeno-associated virus, hepatocellular carcinoma, bone marrow transplantation
Journal
MOLECULAR THERAPY
Volume -, Issue -, Pages -
Publisher
Elsevier BV
Online
2021-01-01
DOI
10.1016/j.ymthe.2020.12.031
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- MAGI1, a New Potential Tumor Suppressor Gene in Estrogen Receptor Positive Breast Cancer
- (2020) Begoña Alday-Parejo et al. Cancers
- Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology
- (2020) Juliette Hordeaux et al. HUMAN GENE THERAPY
- TMEM166 inhibits cell proliferation, migration and invasion in hepatocellular carcinoma via upregulating TP53
- (2020) Jiejie Yang et al. MOLECULAR AND CELLULAR BIOCHEMISTRY
- A HILIC-MS/MS method for simultaneous quantification of the lysosomal disease markers galactosylsphingosine and glucosylsphingosine in mouse serum
- (2018) Rohini Sidhu et al. BIOMEDICAL CHROMATOGRAPHY
- AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease)
- (2018) Allison M. Bradbury et al. HUMAN GENE THERAPY
- Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe’s Disease after Global Gene Therapy
- (2018) Michael S. Marshall et al. MOLECULAR THERAPY
- Low levels of tumor suppressor candidate 3 predict poor prognosis of patients with hepatocellular carcinoma
- (2018) Xu-Ren Sheng et al. OncoTargets and Therapy
- EVA1A inhibits GBM cell proliferation by inducing autophagy and apoptosis
- (2017) Xue Shen et al. EXPERIMENTAL CELL RESEARCH
- Developmental outcomes of cord blood transplantation for Krabbe disease
- (2017) Matthew D. Wright et al. NEUROLOGY
- Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe‐affected non‐human primates by intracerebral lentiviral gene therapy
- (2016) Vasco Meneghini et al. EMBO Molecular Medicine
- Krabbe disease: One Hundred years from the bedside to the bench to the bedside
- (2016) David A. Wenger et al. JOURNAL OF NEUROSCIENCE RESEARCH
- Intrathecal administration of AAV/GALC vectors in 10-11-day-old twitcher mice improves survival and is enhanced by bone marrow transplant
- (2016) Subha Karumuthil-Melethil et al. JOURNAL OF NEUROSCIENCE RESEARCH
- Neuroimmune mechanisms in Krabbe's disease
- (2016) Gregory B. Potter et al. JOURNAL OF NEUROSCIENCE RESEARCH
- Treatment for Krabbe's disease: Finding the combination
- (2016) Christina R. Mikulka et al. JOURNAL OF NEUROSCIENCE RESEARCH
- Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy?
- (2016) Randy J Chandler et al. MOLECULAR THERAPY
- Prognostic value of decreased FOXP1 protein expression in various tumors: a systematic review and meta-analysis
- (2016) Jian Xiao et al. Scientific Reports
- Protein Kinase A-induced tamoxifen resistance is mediated by anchoring protein AKAP13
- (2015) Cristiane Bentin Toaldo et al. BMC CANCER
- Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy
- (2015) A. Ricca 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
- Mechanism-Based Combination Treatment Dramatically Increases Therapeutic Efficacy in Murine Globoid Cell Leukodystrophy
- (2015) J. A. Hawkins-Salsbury et al. JOURNAL OF NEUROSCIENCE
- Long-term Improvements in Lifespan and Pathology in CNS and PNS After BMT Plus One Intravenous Injection of AAVrh10-GALC in Twitcher Mice
- (2015) Mohammad A Rafi et al. MOLECULAR THERAPY
- Long-Term Correction of Sandhoff Disease Following Intravenous Delivery of rAAV9 to Mouse Neonates
- (2015) Jagdeep S Walia et al. MOLECULAR THERAPY
- Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates
- (2015) Kathrin Meyer et al. MOLECULAR THERAPY
- Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas
- (2015) Jean-Charles Nault et al. NATURE GENETICS
- The genome-wide effects of ionizing radiation on mutation induction in the mammalian germline
- (2015) Adeolu B. Adewoye et al. Nature Communications
- Experimental Therapies in the Murine Model of Globoid Cell Leukodystrophy
- (2014) Yedda Li et al. PEDIATRIC NEUROLOGY
- Innate Immune Activation in the Pathogenesis of a Murine Model of Globoid Cell Leukodystrophy
- (2013) Eric R. Snook et al. AMERICAN JOURNAL OF PATHOLOGY
- Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates
- (2013) S J Gray et al. GENE THERAPY
- The galactocerebrosidase enzyme contributes to maintain a functional neurogenic niche during early post-natal CNS development
- (2012) Sara Santambrogio et al. HUMAN MOLECULAR GENETICS
- Prognostic significance of FOXP1 as an oncogene in hepatocellular carcinoma
- (2012) Yixin Zhang et al. JOURNAL OF CLINICAL PATHOLOGY
- Induction of hepatocellular carcinoma by in vivo gene targeting
- (2012) P.-R. Wang et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?
- (2011) J. A. Hawkins-Salsbury et al. HUMAN MOLECULAR GENETICS
- Downregulation of MAGI1 Associates with Poor Prognosis of Hepatocellular Carcinoma
- (2011) Gewen Zhang et al. JOURNAL OF INVESTIGATIVE SURGERY
- Bone Marrow Transplantation Augments the Effect of Brain- and Spinal Cord-Directed Adeno-Associated Virus 2/5 Gene Therapy by Altering Inflammation in the Murine Model of Globoid-Cell Leukodystrophy
- (2011) A. S. Reddy et al. JOURNAL OF NEUROSCIENCE
- Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection
- (2008) Carmela Zincarelli et al. MOLECULAR THERAPY
Find the ideal target journal for your manuscript
Explore over 38,000 international journals covering a vast array of academic fields.
SearchAsk a Question. Answer a Question.
Quickly pose questions to the entire community. Debate answers and get clarity on the most important issues facing researchers.
Get Started