RARE MONOGENIC DISORDERS

THERAPY
  ◊ Gene therapy strategies for rare monogenic disorders with nuclear or mitochondrial gene mutations

GENE THERAPY
  ◊ Gene therapy for ultrarare diseases: a geneticist’s perspective

RED BLOOD CELL DISEASES

GENE EDITING
  ◊ Gene Editing for Inherited Red Blood Cell Disease

RETINA ANGIOGENESIS

CRISPR-Cas9 THERAPY
  ◊Genome editing abrogates angiogenesis in vivo

RETINITIS PIGMENTOSA

CRISPR-Cas9 THERAPY
  ◊Restoration of RPGR expression in vivo using CRISPR/Cas9 gene edition
  ◊Gene Therapy for Retinitis Pigmentosa: Current Challenges and New Progress

RETINOBLASTOMA

NOVEL THERAPY
  ◊SYK

RETINOPATHY

CNG CHANNEL-RELATED RETINOPATHIES
  ◊ Biology, Pathobiology and Gene Therapy of CNG Channel-Related Retinopathies

PEPTIDE THERAPY
  ◊ Peptide suppresses ocular neovascularization and vascular leakage
  ◊ Foxp3 Tregs repair pathological angiogenesis

GENOME-EDITING
  ◊ Genome-Editing Strategies for Treating Human Retinal Degenerations

GENE THERAPY
  ◊ Optogenetic Retinal Gene Therapy with the Light Gated GPCR Vertebrate Rhodopsin
  ◊ Knockdown of VEGFR2 in retinal endothelial cells to treat retinopathy
  ◊ Gene therapy in retinal diseases
  ◊ Clinical and Ocular Inflammatory Inhibitors of Viral-Based Gene Therapy of the Retina

ORGANOIDS
  ◊ Retinal Ciliopathies and Potential Gene Therapies: A Focus on Human iPSC-Derived Organoid Models

RISK
  ◊ ERA; The science and practice of current environmental risk assessment for gene therapy RETT SYNDROME

NOVEL THERAPY
  ◊ Xi reactivation for Rett syndrome and other X-linked disorders
  ◊ Inhibition of Gsk3b reduces NFkappa;-B signaling and rescues synaptic activity to improve the Rett syndrome
  ◊ State-of-the-art therapies for Rett syndrome

REVERSE ALVEOLAR IMMUNODEFICIENCY

IMMUNOTHERAPY
  ◊Macrophages expressing IFN-γ

RHEUMATOID ARTHRITIS

IMMUNOTHERAPY
  ◊ 4-1BB
  ◊ Approaches

THERAPEUTIC STRATEGIES
  ◊ Ncf1
  ◊ Synoviolin

THERAPY MURINE MODEL
  ◊ Cryptic epitope SLAYGLR in osteopontin

RNA THERAPEUTICS

NEW APPROACHES
  ◊ Advances
  ◊ Advances
  ◊ Systemic administration of modified siRNAs
  ◊ RNA-Based Therapy Do for Monogenic Diseasess
  ◊ RNA editing: Expanding the potential of RNA therapeutics
  ◊ RNA-targeting strategies as a platform for ocular gene therapy