Gene Therapy in Mouse Models of Huntington Disease

Perturbation with Intrabodies Reveals That Calpain Cleavage Is Required for Degradation of Huntingtin Exon 1

(2011) Amber L. Southwell et al.   PLoS One

Complexity and Heterogeneity: What Drives the Ever-changing Brain in Huntington's Disease?

(2010) H. Diana Rosas et al.   Annals of the New York Academy of Sciences

Genetically engineered mesenchymal stem cells reduce behavioral deficits in the YAC 128 mouse model of Huntington's disease

(2010) Nicholas D. Dey et al.   BEHAVIOURAL BRAIN RESEARCH

Ex vivo delivery of GDNF maintains motor function and prevents neuronal loss in a transgenic mouse model of Huntington's disease

(2010) Allison D. Ebert et al.   EXPERIMENTAL NEUROLOGY

BDNF regulation under GFAP promoter provides engineered astrocytes as a new approach for long-term protection in Huntington's disease

(2010) A Giralt et al.   GENE THERAPY

Rilmenidine attenuates toxicity of polyglutamine expansions in a mouse model of Huntington's disease

(2010) C. Rose et al.   HUMAN MOLECULAR GENETICS

Expression of Huntington's disease protein results in apoptotic neurons in the brains of cloned transgenic pigs

(2010) Dongshan Yang et al.   HUMAN MOLECULAR GENETICS

An ovine transgenic Huntington's disease model

(2010) J. C. Jacobsen et al.   HUMAN MOLECULAR GENETICS

Early or Late-Stage Anti-N-Terminal Huntingtin Intrabody Gene Therapy Reduces Pathological Features in B6.HDR6/1 Mice

(2010) Abigail Snyder-Keller et al.   JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY

Harnessing chaperone-mediated autophagy for the selective degradation of mutant huntingtin protein

(2010) Peter O Bauer et al.   NATURE BIOTECHNOLOGY

Molecular Mechanisms and Potential Therapeutical Targets in Huntington's Disease

(2010) Chiara Zuccato et al.   PHYSIOLOGICAL REVIEWS

Sustained effects of nonallele-specificHuntingtinsilencing

(2009) Valérie Drouet et al.   ANNALS OF NEUROLOGY

Allele-Selective Inhibition of Mutant Huntingtin by Peptide Nucleic Acid-Peptide Conjugates, Locked Nucleic Acid, and Small Interfering RNA

(2009) Jiaxin Hu et al.   Annals of the New York Academy of Sciences

Progress and Challenges in RNA Interference Therapy for Huntington Disease

(2009) Scott Q. Harper   ARCHIVES OF NEUROLOGY

Mitochondria in Huntington's disease

(2009) Maria Damiano et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Five siRNAs Targeting Three SNPs May Provide Therapy for Three-Quarters of Huntington's Disease Patients

(2009) Edith L. Pfister et al.   CURRENT BIOLOGY

A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference

(2009) Maria Stella Lombardi et al.   EXPERIMENTAL NEUROLOGY

Human α-Iduronidase Gene Transfer Mediated by Adeno-Associated Virus Types 1, 2, and 5 in the Brain of Nonhuman Primates: Vector Diffusion and Biodistribution

(2009) Carine Ciron et al.   HUMAN GENE THERAPY

Striatal Expression of a Calmodulin Fragment Improved Motor Function, Weight Loss, and Neuropathology in the R6/2 Mouse Model of Huntington's Disease

(2009) Y. Dai et al.   JOURNAL OF NEUROSCIENCE

Intrabody Gene Therapy Ameliorates Motor, Cognitive, and Neuropathological Symptoms in Multiple Mouse Models of Huntington's Disease

(2009) A. L. Southwell et al.   JOURNAL OF NEUROSCIENCE

Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice

(2009) Ryan L Boudreau et al.   MOLECULAR THERAPY

Motor abnormalities in premanifest persons with Huntington's disease: The PREDICT-HD study

(2009) Kevin M. Biglan et al.   MOVEMENT DISORDERS

Safety and tolerability of putaminal AADC gene therapy for Parkinson disease

(2009) C. W. Christine et al.   NEUROLOGY

Huntington's disease: from pathology and genetics to potential therapies

(2008) Sara Imarisio et al.   BIOCHEMICAL JOURNAL

Huntington’s disease: degradation of mutant huntingtin by autophagy

(2008) Sovan Sarkar et al.   FEBS Journal

Suppression of neuropil aggregates and neurological symptoms by an intracellular antibody implicates the cytoplasmic toxicity of mutant huntingtin

(2008) Chuan-En Wang et al.   JOURNAL OF CELL BIOLOGY

A novel pathogenic pathway of immune activation detectable before clinical onset in Huntington's disease

(2008) Maria Björkqvist et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Intrabodies Binding the Proline-Rich Domains of Mutant Huntingtin Increase Its Turnover and Reduce Neurotoxicity

(2008) A. L. Southwell et al.   JOURNAL OF NEUROSCIENCE

Sustained striatal ciliary neurotrophic factor expression negatively affects behavior and gene expression in normal and R6/1 mice

(2008) Eileen M. Denovan-Wright et al.   JOURNAL OF NEUROSCIENCE RESEARCH

Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2–neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial

(2008) William J Marks et al.   LANCET NEUROLOGY

AAV Vector–mediated RNAi of Mutant Huntingtin Expression Is Neuroprotective in a Novel Genetic Rat Model of Huntington's Disease

(2008) Nicholas R Franich et al.   MOLECULAR THERAPY

Towards a transgenic model of Huntington’s disease in a non-human primate

(2008) Shang-Hsun Yang et al.   NATURE

Intrastriatal CERE-120 (AAV-Neurturin) protects striatal and cortical neurons and delays motor deficits in a transgenic mouse model of Huntington's disease

(2008) Shilpa Ramaswamy et al.   NEUROBIOLOGY OF DISEASE

Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi

(2008) J. L. McBride et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA