НАСЛЕДСТВЕННЫЕ БОЛЕЗНИ
ТЕРАПИЯ

FABRY DISEASE
  • GENE THERAPY
      ◊ Gene therapy for Fabry disease and outlooks on gene-editing

    • FANCONI ANEMIA
  • BASE EDITING
      ◊ Adenine base editing is an efficient approach to restore function in FA patient cells

    • FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
  • DRUG THERAPY
      ◊ beta-2 adrenergic receptor inhibit DUX4 expression

    • FAMILIAL AMYOTROPHIC LATERAL SCLEROSIS
  • FUNCTION
      ◊ FALS and copper

    • FAMILIAL DYSAUTONOMIA (FD)
  • SPLICING DEFECT IN IKBKAP
      ◊ Antisense oligonucleotides correct splicing defect in IKBKAP transgenic mice

    • FAMILIAL HYPERCHOLESTEROLEMIA
  • GENE THERAPY
      ◊ Long Noncoding RNA Facilitated Gene Therapy
      ◊ Construction of optimised cassette for gene therapy of familial hypercholesterolemia: bicistronic vector
      ◊ Gene Therapy Targeting PCSK9

    • FETAL ANOMALIES
  • GENE THERAPY
      ◊ in utero gene therapy: advances, challenges, and future considerations

    • FIBRILLATION
  • ATRIAL
      ◊ Factor Xa inhibitors

    • FIBROSIS DISEASES
  • FIBROCARTILAGE
      ◊ Articular fibrocartilage-targeted therapy by microtubule stabilization

  • GENE THERAPY
      ◊ Relaxin gene therapy

    • FRAGILE X SYNDROME
  • GENE EDITING
      ◊Rescue of Fragile X Syndrome Neurons by DNA Methylation Editing of the FMR1 Gene