HAEMATOPOIETIC STEM CELL

CELL and GENE THERAPY
  ◊ Immunological barriers to haematopoietic stem cell gene therapy
  ◊ Base Editors-Mediated Gene Therapy in Hematopoietic Stem Cells for Hematologic Diseases

HAEMATOPOIETIC SYSTEM DISORDERS

THERAPEUTICS STRATEGIES
  ◊ Genome and Epigenome Editing

GENE THERAPY
  ◊ Genetic engineering of hematopoiesis
  ◊ Gene Editing of Hematopoietic Stem Cells
  ◊ Gene therapy using haematopoietic stem and progenitor cells
  ◊ Homology-directed gene-editing approaches for hematopoietic stem and progenitor cell gene therapy
  ◊ CRISPR/Cas9-mediated gene editing. A promising strategy in hematological disorders
  ◊ Ultra-deep sequencing validates safety of CRISPR/Cas9 genome editing in human hematopoietic stem and progenitor cells

β-HEMOGLOBINOPATHIES
  ◊ lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial
  ◊ CRISPR/Cas9-based multiplex genome editing of BCL11A and HBG efficiently induces fetal hemoglobin expression
  ◊ Gene therapy approaches to promote fetal hemoglobin production for the treatment of β-hemoglobinopathies
  ◊Autologous gene therapy for hemoglobinopathies: From bench to patient’s bedside

HEARING LOSS

FIXING GENE DEFECTS
  ◊ Nanoparticles

GENE THERAPY
  ◊ Engraftment of Human Pluripotent Stem Cell-derived Progenitors
  ◊ Fetal gene therapy to treat congenital hearing loss and vestibular dysfunction

  ◊ gene editing of outer hair cells prevents progressive hearing loss in a dominant-negative KCNQ4 murine model
  ◊ genome editing for genetic hearing loss
  ◊ Adeno-associated viral inner-ear gene therapy for sensorineural hearing loss

CELL THERAPY
  ◊ Otoferlin linking SNAREs and calcium channels

HEART DISEASE

THERAPEUTIC ANGIOGENESIS
  ◊Angiogenic Factor AGGF1
  ◊Coronary arteries and Collateral formation

HEART FAILURE

BEATING CONGENITAL HEART DEFECTS
  ◊ embryonic stem cells

GENE THERAPY
  ◊ Design of a Phase 3 trial of intracoronary administration of human adenovirus 5 encoding human adenylyl cyclase type 6 (RT-100) gene transfer in patients with heart failure with reduced left ventricular ejection fraction
  ◊ Advanced cell and gene therapies in cardiology

INFARCTED HEARTS
  ◊ Mesenchymal stem cells

MICROTUBULES
  ◊ Suppression of detyrosinated microtubules improves cardiomyocyte function in human heart failure

NORMALIZING CALCIUM CYCLING
  ◊ Therapeutic approaches

STATINS
  ◊ Therapeutic implications of statins in heart failure with reduced ejection fraction

HEMOPHILIA

GENE THERAPY
  ◊ Hemophilia Gene Therapy: The End of the Beginning?

HEMOPHILIA A
  ◊ Modulation of Immune Reaction in Hydrodynamic Gene Therapy for Hemophilia A
  ◊ Hemophilia A gene therapy: current and next-generation approaches

HEMOPHILIA ARTHROPATHY
  ◊ Intra-articular adeno-associated virus-mediated gene therapy for hemophilia arthropathy

HEMOPHILIA B

THERAPEUTICS STRATEGIES
  ◊ Immune Modulatory Cell Therapy after Gene Transfer
  ◊ Gene Therapy for Hemophilia
  ◊ Targeted genome engineering in human induced pluripotent stem cells from patients with hemophilia B
  ◊ How to translate and implement the current science of gene therapy into haemophilia care?
  ◊ Hemophilia B and gene therapy: a new chapter with etranacogene dezaparvovec

HEPATOCYTES

GENE EDITING
  ◊ Ex Vivo/In vivo Gene Editing in Hepatocytes HEPATITIS C

THERAPEUTICS STRATEGIES
  ◊ : current status
  ◊ SPP inhibitors suppressing propagation of HCV

HEPATITIS B
  ◊ : Progress and Future Prospective

HEPATOCELLULAR CARCINOMA

INHIBITING ALTERNATIVE ACTIVATION OF TUMOR-ASSOCIATED MACROPHAGEs Estrogens

ANTI-CANCER PROTEIN The protein histidine phosphatase LHPP is a tumour suppressor

DELIVERY silence Sox2 gene expression enhances therapeutic response in hepatocellular carcinoma A Lactose-Derived CRISPR/Cas9 Delivery System for Efficient Genome Editing In Vivo to Treat Orthotopic Hepatocellular Carcinoma

CELL THERAPY Adoptive Cell Therapy in Hepatocellular Carcinoma

GENE THERAPY Use of CRISPR/Cas9 gene therapy to treat hepatocellular carcinoma Recent advances in various adeno-associated viruses (AAVs) as gene therapy agents in hepatocellular carcinoma

HEREDITARY TYROSINEMIA TYPE 1

LENTIVIRAL VECTOR GENE THERAPY In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions

HETEROPLASMIC mtDNA DISEASES

THERAPEUTICS STRATEGIES
  ◊DNA-editing enzymes as potential treatments for heteroplasmic mtDNA diseases HYDROCEPHALUS

tumor-associated
  ◊Choroid plexus mast cells drive tumor-associated hydrocephalus HIV-1 INFECTION

DRUGS
  ◊ NRTIs and NNRTIs
  ◊ Enfuvirtide
  ◊ HIV Vaccine

SUPPRESSION RETROVIRAL REPLICATION
  ◊ Integrase inhibitors L-870812

HIV-1 INHIBITOR
  ◊ Targets the HIV-1 envelope and inhibits CD4 receptor binding
  ◊ PSC-RANTES
  ◊ Novel TALEN Targeting CCR5 Confirms Efficacy and Safety in Conferring Resistance to HIV-1 Infection

HIV-1 GENEN THERAPY
  ◊ CCR5 Gene Editing in Hematopoietic Stem and Progenitor Cells for the Treatment of HIV
  ◊ CRISPR-based gene editing in HIV-1/AIDS therapy
  ◊ Advances in cell and gene therapy for HIV disease
  ◊ Advances in HIV Gene Therapy

HUNTINGTON'S DISEASE

CYTOPROTECTION
  ◊ Golgi stress response reprograms cysteine metabolism to confer cytoprotection

DRUGS THERAPEUTICS
  ◊ Decoding the structure of huntingtin

GENE THERAPY
  ◊ Combination of stem cell and gene therapy

  ◊ Conversion of striatal astrocytes into GABAergic neurons in mouse models of Huntingtons disease
  ◊ Gene Therapy for Huntington’s Disease:

EXPERIMENTAL THERAPEUTICS
  ◊ Proteolysis of huntingtin, aggregation of huntingtin
  ◊ RNAi mechanisms in Huntington’s disease therapy
  ◊ Combination of stem cell and gene therapy ameliorates symptoms in Huntington’s disease mice

HUTCHINSON-GILFORD PROGERIA

ENHANCE HEALTHSPAN
  ◊ Targeting of NAT10 enhances healthspan in a mouse model accelerated aging syndrome

HYPERTROPHIC CARDIOMYOPATHY

GENE THERAPY CRISPR
  ◊ Correction of a pathogenic gene mutation in human embryos

HYPERALGESIA

GLIA
  ◊ Novel drug discovery target

HYPERTENSION

GENE THERAPY
  ◊ Genetic Delivery and Gene Therapy in Pulmonary Hypertension

HYPOXIA

HIF-1 AS A TARGET FOR DRUGS
  ◊ : Therapeutic potential

HYPOSOMNIA

THERAPY
  ◊ Melatonin promotes sleep in mice by inhibiting orexin neurons in the perifornical lateral hypothalamus