Deimmunization for gene therapy: host matching of synthetic zinc finger constructs enables long-term mutant Huntingtin repression in mice

Update on Huntington's disease: Advances in care and emerging therapeutic options

(2015) Daniel Zielonka et al.   PARKINSONISM & RELATED DISORDERS

Synthetic biology and therapeutic strategies for the degenerating brain

(2014) Carmen Agustín-Pavón et al.   BIOESSAYS

Unstable expression of transgene is associated with the methylation of CAG promoter in the offspring from the same litter of homozygous transgenic mice

(2014) Yang Zhou et al.   MOLECULAR BIOLOGY REPORTS

Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV

(2014) Pablo Tebas et al.   NEW ENGLAND JOURNAL OF MEDICINE

Pre-Clinical Assessment of Immune Responses to Adeno-Associated Virus (AAV) Vectors

(2014) Etiena Basner-Tschakarjan et al.   Frontiers in Immunology

Convection-enhanced delivery improves distribution and efficacy of tumor-selective retroviral replicating vectors in a rodent brain tumor model

(2013) D Yin et al.   CANCER GENE THERAPY

AAV9-mediated Expression of a Non-self Protein in Nonhuman Primate Central Nervous System Triggers Widespread Neuroinflammation Driven by Antigen-presenting Cell Transduction

(2013) Lluis Samaranch et al.   MOLECULAR THERAPY

Strategies for the Delivery of Multiple Collinear Infusion Clouds in Convection-Enhanced Delivery in the Treatment of Parkinson's Disease

(2013) Karl Sillay et al.   STEREOTACTIC AND FUNCTIONAL NEUROSURGERY

HDAC4 Reduction: A Novel Therapeutic Strategy to Target Cytoplasmic Huntingtin and Ameliorate Neurodegeneration

(2013) Michal Mielcarek et al.   PLOS BIOLOGY

Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression

(2012) Dongbo Yu et al.   CELL

Understanding lentiviral vector chromatin targeting: working to reduce insertional mutagenic potential for gene therapy

(2012) C Papayannakos et al.   GENE THERAPY

Cerebral Infusion of AAV9 Vector-encoding Non-self Proteins Can Elicit Cell-mediated Immune Responses

(2012) Agnieszka Ciesielska et al.   MOLECULAR THERAPY

Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

(2012) Holly B. Kordasiewicz et al.   NEURON

Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice

(2012) M. Garriga-Canut et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Zinc-finger nucleases: how to play two good hands

(2011) Mark Isalan   NATURE METHODS

Optimization algorithms for functional deimmunization of therapeutic proteins

(2010) Andrew S Parker et al.   BMC BIOINFORMATICS

Molecular Mechanisms and Potential Therapeutical Targets in Huntington's Disease

(2010) Chiara Zuccato et al.   PHYSIOLOGICAL REVIEWS

KRAB–Zinc Finger Proteins and KAP1 Can Mediate Long-Range Transcriptional Repression through Heterochromatin Spreading

(2010) Anna C. Groner et al.   PLoS Genetics

Transduction of Nonhuman Primate Brain with Adeno-Associated Virus Serotype 1: Vector Trafficking and Immune Response

(2009) Piotr Hadaczek et al.   HUMAN GENE THERAPY

Genetic Knock-Down of HDAC7 Does Not Ameliorate Disease Pathogenesis in the R6/2 Mouse Model of Huntington's Disease

(2009) Caroline L. Benn et al.   PLoS One