НАСЛЕДСТВЕННЫЕ БОЛЕЗНИ
ТЕРАПИЯ


B


B-CELL LEUCOSIS AND LYMPHOMS
  • GENE THERAPY
      ◊ gene therapy to achieve long-term continuous or controllable expression of biotherapeutics
  • CELL THERAPY
      ◊ Non-viral, specifically targeted CAR-T cells achieve high safety and efficacy in B-NHL


  • B CELL-BASED GENE THERAPIES
  • GENE THERAPY
      ◊ Vector Strategies to Actualize B Cell-Based Gene Therapies


  • BASE EDITING DNA
  • ZINC FINGER DEAMINASES
      ◊Nuclear and mitochondrial DNA editing in human cells with zinc finger deaminases
  • CHOLESTEROL
      ◊Super-precise gene-editing approach switches off a gene in the liver that regulates ‘bad’ cholesterol.
  • BASE EDITING mtDNA
  • TALE-LINKED DEAMINASES
      ◊Engineering TALE-linked deaminases to facilitate precision adenine base editing in mitochondrial DNA BATTEN DISEASE
  • CURRENT TREATMENTS
      ◊ S Therapeutic landscape for Batten disease
  • β-THALASSEMIA
  • STEM CELL GENE THERAPY
      ◊ S Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent β-thalassemia
  • TRANSFUSION-DEPENDENT β-THALASSEMIA
      ◊ Approach for Transfusion-Dependent β-Thalassemia—CRISPR-Cas9 Gene Editing for β-Thalassemia
  • TARGETED THERAPY
  • TARGETED BIOLOGICS
      ◊ Targeted Biologics: The New Frontier for Precision Therapy
  • BILIARY DISEASES
  • BILIARY FIBROSIS
      ◊ S Current therapies and novel approaches for biliary diseases
  • 3D BIOPRINTING
  • MODULATION FOR GENETIC EXPRESSION
      ◊ S 3D Printed Bioconstructs: Regenerative Modulation for Genetic Expression
  • BLOOD DISEASES
  • GENE THERAPY
      ◊ S Gene therapy for blood diseases
      ◊ SGene therapy and gene editing strategies in inherited blood disorders

  •   ◊ S Advances in the gene therapy of monogenic blood cell diseases
      ◊ S On gene therapy and gene editing for sickle cell disease and hemophilia
    BLOOD SUBSTITUTES
  • OXYGEN THERAPEUTICS
      ◊ SSupressing the 'radical' nature of Hb