Read the Special Issue: Rational Design of Gene Therapy Vectors

Can't see this email properly? [Click here to view an online version]( [Molecular Therapy]( [American Society of Gene & Cell Therapy]( Special Issue: Rational Design of Gene Therapy Vectors [View the Special Issue]( In this special issue of Molecular Therapy - Methods & Clinical Development, which is on "Rational Design of Gene Therapy Vectors," seven review articles highlight current efforts to improve the efficacy of gene therapy. Delivery tools and transgene expression cassettes are being designed and optimized for the treatment of diseases ranging from rare genetic disorders to major public health concerns, such as cancer. Editorial - [Rational Design of Gene Therapy Vectors]( Reviews - [Engineering Nanoparticles for Targeted Delivery of Nucleic Acid Therapeutics in Tumor]( - [Surface-Engineered Lentiviral Vectors for Selective Gene Transfer into Subtypes of Lymphocytes]( - [Engineering Globin Gene Expression]( - [Engineering and Design of Chimeric Antigen Receptors]( - [Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy]( - [Transcriptional Targeting and MicroRNA Regulation of Lentiviral Vectors]( - [Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors]( Original Articles - [Efficient Induction of T Cells against Conserved HIV-1 Regions by Mosaic Vaccines Delivered as Self-Amplifying mRNA]( - [Efficacy of a Bicistronic Vector for Correction of Sandhoff Disease in a Mouse Model]( - [Shifting Retroviral Vector Integrations Away from Transcriptional Start Sites via DNA-Binding Protein Domain Insertion into Integrase]( - [Codon Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality]( - [AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice]( - [A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing]( - [Preproinsulin Designer Antigens Excluded from Endoplasmic Reticulum Suppressed Diabetes Development in NOD Mice by DNA Vaccination]( - [Development of a Novel Anti-CD19 Chimeric Antigen Receptor: A Paradigm for an Affordable CAR T Cell Production at Academic Institutions]( - [Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome]( - [TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction]( - [Novel Chimeric Gene Therapy Vectors Based on Adeno-Associated Virus and Four Different Mammalian Bocaviruses]( - [Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease]( [View the Special Issue]( This message has been sent to {EMAIL} from Elsevier Communications on behalf of Cell Press. If you no longer wish to receive messages of this nature from us in the future, please [click here](. Visit the [Elsevier Preference Center]( to manage more of your communication preferences with us. Copyright © 2019 [Elsevier B.V.](. All rights reserved. | [Elsevier Privacy Policy]( Elsevier B.V. Registered Office: Radarweg 29, 1043 NX Amsterdam, The Netherlands. Reg. No. 33158992 – Netherlands. VAT No. NL 005033019B01.