Volume 25, Issue 7, Pages (July 2017)

Slides:



Advertisements
Similar presentations
Volume 9, Issue 3, Pages (March 2004)
Advertisements

Volume 12, Issue 5, Pages (November 2005)
Volume 10, Issue 2, Pages (August 2004)
Volume 20, Issue 2, (February 2012)
Production and clinical development of nanoparticles for gene delivery
209. Use of Helicobacter pyroli Neutrophil Activating Protein (NAP) as an Immune- Modulatory Agent To Enhance the Efficacy of Oncolytic Adenovirus Therapy.
Molecular targeted therapies in hepatocellular carcinoma: From pre-clinical models to clinical trials  Pippa Newell, Augusto Villanueva, Josep M. Llovet 
Engineering Natural Killer Cells for Cancer Immunotherapy
Genome-editing Technologies for Gene and Cell Therapy
Molecular Therapy - Nucleic Acids
627. Non-Invasive, Multimodal Imaging of Microvesicles with Metabolically Biotinylated, Membrane-Bound Gaussia Luciferase    Molecular Therapy  Volume.
Will Engineered T Cells Expressing CD20 scFv Eradicate Melanoma?
162. Stability of Polymer/Plasmid DNA Complexes In Vitro and In Vivo
281. Rapid Generation of Induced Pluripotent Stem Cells (iPSCs) from the Urine of a Patient with Duchenne Muscular Dystrophy    Molecular Therapy  Volume.
Volume 25, Issue 7, Pages (July 2017)
Molecular Therapy  Volume 20, Pages S261-S262 (May 2012) DOI: /S (16)
Evolving Gene Therapy in Primary Immunodeficiency
In Vivo Gene Delivery by Nonviral Vectors: Overcoming Hurdles?
Jiehua Zhou, John J. Rossi  Molecular Therapy - Nucleic Acids 
343. Benchtop DNA Synthesizer: Oligo-Templated Polymerization (OTP)
Engineering Natural Killer Cells for Cancer Immunotherapy
637. Effect of PEG Grafting Degree on Morphology and Transfection Efficiency of Polycation-g-PEG/DNA Nanoparticles    Molecular Therapy  Volume 20, (May.
John R. Martin, Christopher E. Nelson, Mukesh K
Volume 26, Issue 2, Pages (February 2018)
HIV Receives a “One Two Knockout Punch”
Volume 25, Issue 1, Pages 5-7 (January 2017)
Lipid Nanoparticle Systems for Enabling Gene Therapies
Differential conjugation of endogenous SUMO-1 and endogenous SUMO-2/3 to target proteins.A and B, SUMO-1 and SUMO-2 proteins were produced in E coli and.
Volume 25, Issue 5, Pages (May 2017)
The Other Face of Chimeric Antigen Receptors
Volume 9, Issue 3, Pages (March 2004)
724. Blood-Derived Endothelial Progenitor Cells Can Be Isolated With a Novel Method of Diluted Whole Blood Incubation with AMD3100-Induced Mobilization.
Genome-editing Technologies for Gene and Cell Therapy
Valder R Arruda, Patricia Favaro, Jonathan D Finn  Molecular Therapy 
Premal Lulla, Carlos A. Ramos  Molecular Therapy 
Molecular Therapy  Volume 20, (May 2012) DOI: /S (16)
Molecular Therapy  Volume 7, Issue 5, (May 2003) DOI: /S (16)
547. AAV Preparations Contain Contamination from DNA Sequences in Production Plasmids Directly Outside of the ITRs  Mark A. Brimble, Junfang Zhou, Christopher.
Knocking down Disease with siRNAs
847. Eradication of Therapy-Resistant Human Prostate Tumors Using an Ultrasound Guided Site-Specific Cancer Terminator Virus Delivery Approach    Molecular.
Volume 26, Issue 4, Pages (April 2018)
Designer Lipids Advance Systemic siRNA Delivery
Volume 25, Issue 5, Pages (May 2017)
133. Survival with Normal Neurological Development of the Juvenile Lethal Urea Cycle Defect Arginase Deficient Mouse with AAV Gene Therapy    Molecular.
660. Bowel/Bladder Sensation and Control in Patients with Spinal Cord Injury Treated with Human Embryonic Stem Cell Therapy  Geeta Shroff  Molecular Therapy 
Volume 26, Issue 2, Pages (February 2018)
521. Multiplex Genome Editing of TCR°/CD52 Genes as a Platform for “Off the Shelf” Adoptive T-Cell Immunotherapies    Molecular Therapy  Volume 22, Pages.
335. High Content Analysis Platform for Optimization of Lipid Mediated CRISPR-Cas9 Delivery Strategies in Human Cells  Benjamin Steyer, Jared Carlson-Stevermer,
405. Hindlimb and Forelimb Strength Assessment in a Canine Model of X-Linked Myotubular Myopathy Following AAV-Mediated Gene Replacement    Molecular.
Live and Let Die: A New Suicide Gene Therapy Moves to the Clinic
Volume 26, Issue 4, Pages (April 2018)
674. Molecular, Biochemical and Biomechanical Analysis of Articular Cartilage Repaired with Genetically Modified Chondrocytes Expressing Insulin-Like.
Chimeric antigen receptor T-cell therapy for solid tumors
Molecular Therapy  Volume 18, Pages S260-S261 (May 2010) DOI: /S (16)
In This Issue Molecular Therapy Volume 16, Issue 4, (April 2008)
Volume 8, Issue 1, Pages (July 2003)
521. AAV Immunotherapy Induces Functional Antigen Specific Regulatory T-Cells to a Neuroantigen: A Potential Treatment for MS  Brad E. Hoffman  Molecular.
Rajendra N. Mitra, Min Zheng, Zongchao Han
773. Detection of RNA Interference (RNAi) Mediated mRNA Cleavage in Fresh Injected Tumor Tissue from Patients in a Phase I Trial of pbi-shRNA™ Lipoplex.
740. Prevention of Radiation-Induced Lung Injury by Administration of Gene-Modified Mesenchymal Stem Cells    Molecular Therapy  Volume 20, Pages S285-S286.
Molecular Therapy  Volume 21, Pages S247-S248 (May 2013)
Vanadium: A Panacea for Resistance to Oncolytic Immunotherapy?
MicroRNA-21 and the Vulnerability of Atherosclerotic Plaques
86. A Highly Compact Epitope-Based Marker Suicide Gene for Safer and Easier Adoptive T-Cell Gene Therapy    Molecular Therapy  Volume 20, Pages S35-S36.
Hisataka Kobayashi, Peter L. Choyke  Kidney International 
Yun Zhang, Felix Schlachetzki, William M Pardridge  Molecular Therapy 
Sensing Bad: Are Co-stimulatory CAR-Expressing γδ T Cells Safer?
Jiehua Zhou, John J. Rossi  Molecular Therapy - Nucleic Acids 
Shirley Wong, Roderick A. Slavcev
624. Randomized Phase II Trial of Adjuvant Autologous Tumor Cell Vaccine (FANG™) for High Risk Stage III/IV Ovarian Cancer: Preliminary Results    Molecular.
Presentation transcript:

Volume 25, Issue 7, Pages 1491-1500 (July 2017) Current Progress in Non-viral RNAi-Based Delivery Strategies to Lymphocytes  Shoshy Mizrahy, Inbal Hazan-Halevy, Niels Dammes, Dalit Landesman-Milo, Dan Peer  Molecular Therapy  Volume 25, Issue 7, Pages 1491-1500 (July 2017) DOI: 10.1016/j.ymthe.2017.03.001 Copyright © 2017 The American Society of Gene and Cell Therapy Terms and Conditions

Figure 1 RNAi Delivery Strategies Available for Lymphocytes (A) SNALP coated with specific ligand molecules encapsulated with siRNAs. (B) Apt-siRNA conjugate. (C) Cationic polymer-based nanoparticles encapsulated with plasmid DNA and siRNAs. (D) Single-chain variable fragment (scFv)-siRNA conjugate. (E) Antibody-protamine fusion protein-siRNA conjugate. Molecular Therapy 2017 25, 1491-1500DOI: (10.1016/j.ymthe.2017.03.001) Copyright © 2017 The American Society of Gene and Cell Therapy Terms and Conditions

Figure 2 Schematic Diagram of the Production Process of Targeted LNPs Lipids including the ionizable lipid and PEG-maleimide dissolved in ethanol and siRNAs in acetate buffer are combined by the microfluidic mixer to produce malamide-functionalized LNPs. Reduced immunoglobulin Gs (IgGs) are conjugated with maleimide-functionalized LNPs to produce targeted LNPs (tLNPs). To remove unconjugated antibody, tLNPs are purified by gel filtration chromatography. mAb, monoclonal antibody. Molecular Therapy 2017 25, 1491-1500DOI: (10.1016/j.ymthe.2017.03.001) Copyright © 2017 The American Society of Gene and Cell Therapy Terms and Conditions