НАСЛЕДСТВЕННЫЕ БОЛЕЗНИ
ТЕРАПИЯ



G


G PROTEIN-COUPLED RECEPTORS
  • MUTATIONS Mechanisms, Pathophysiology and Potential Therapeutic Approaches
    GASTRULATION
  • SINGLE-CELL RESOLUTIONSINGLE-CELL RESOLUTION Multi-omics profiling of mouse gastrulation at single-cell resolution
    GASTROINTESTINAL TUMORS
  • COMBINING THERAPY Nanoparticles Combining Gene Therapy and Chemotherapy as a Treatment for Gastrointestinal Tumors
    neuronopathic GAUCHER DISEASE
  • GENE THERAPIES Fetal gene therapy for neurodegenerative disease of infants Top Five Gene Therapy Stories of 2019 Principles of and strategies for germline gene therapy CRISPR/Cas precise genome editing technology for gene therapy and crop improvement From preclinical development to clinical application Gene therapy from Brazil, South Africa, and India Four Success Stories in Gene Therapy Twisted DNA Increases CRISPR Off-target Effects
  • THERAPIES Advancements in Viral Gene Therapy for Gaucher Disease
  • GENE CORRECTION
  • DONOR-FREE CORRECTION Gene correction by targeted interhomolog recombination
  • GENE EDITION
  • OPTIMIZED CRISPR-Cas9 COMPONENTS Integrating gene delivery and gene-editing technologies CRISPR/Cas-Dependent and Nuclease-Free In Vivo Therapeutic Gene Editing Human Genome Editing: What Are the Rules? CRISPR-Stop Codon Read Through method to control Cas9 expression for gene editing Next-Generation Tools for Gene Editin Avoiding Gene Editing’s Unintended Consequences
  • GENE THERAPY
  • ANTISENSE OLIGONUCLEOTIDES Antisense oligonucleotides and other genetic therapies
  • GENOTOXIC EFFECTS Genotoxic Effects of Base and Prime Editing
  • PROGRAMMABLE SYNTHETIC RECEPTORS Programmable synthetic receptors: the next-generation of cell and gene therapies
  • gRNA Selection of extended CRISPR RNAs with enhanced targeting and specificity
  • TRIALS Record number of gene-therapy trials, despite setbacks
  • DURABILITY OF TRANSGENE EXPRESSION Durability of transgene expression after rAAV gene therapy
  • GENE THERAPY SUICIDE
  • APPROACHES Origins of Suicide Gene Therapy
  • DELIVERY In vivo methods for acute modulation of gene expression in the central nervous system
    A Universal GSH-Responsive Nanoplatform for the Delivery of DNA, mRNA, and Cas9/sgRNA
    Ophthalmic Administration of a DNA Plasmid Harboring the Murine Tph2 Gene
    Cyclodextrins in drug delivery
    Cyclodextrins in drug delivery
    New methods for effective transport of large genes in gene therapy
  • GENE EDITING CRISPR-cas gene-editing as plausible treatment of neuromuscular and nucleotide-repeat-expansion diseases
  • GENE THERAPY FETAL Gene and Stem Cell Therapies for Fetal Care
  • EX VIVO Genetic diseases and leukemia Gene therapy with transposons
  • IN HUMANs CRISPR Genome Editing and Gene Therapy HIV


  • IN UTERO In Utero Gene Therapy and Genome Editing
  • PROGRESS, PROBLEMS, VIRAL VECTORS Advances in pigs using CRISPR-Cas9 Gene Therapy With Regulatory T Cells CRISPR/Cas9: A revolution in genetic disease treatment Knockdown of Human AMPK Using the CRISPR/Cas9 Genome-Editing CRISPR Keeps Things Fresh: Next-Generation Tools for Gene Editing Next-Generation Tools for Gene Editing Gene Therapy in ALS and SMA: Advances, Challenges and Perspectives


  • GENE REPAIR
  • DEVELOPMENT AND REGULATION Advances