AROMATIC L-AMINO ACID DECARBOXYLASE DEFICIENCY

GENE THERAPY
  ◊ Gene therapy for aromatic L-amino acid decarboxylase deficiency: Requirements for safe application and knowledge-generating follow-up

ACHROMATOPSIA

GENE THERAPY
  ◊ Gene Therapy for the Treatment of Achromatopsia


  ◊ Gene Therapy for Achromatopsia

ACUTE MYELOID LEUKEMIA (AML)

THERAPY
  ◊ Activation of BAX by BTSA1 overcomes apoptosis resistance

GENE THERAPY
  ◊ Overcoming Cancer Therapy Obstacles With Epitope Editing

ACUTE NEUROLOGICAL INSULTS

NEUROPROTECTIVE GENE THERAPY
  ◊ Mechanisms

ADENINE BASE EDITORS

GENE THERAPY
  ◊ Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors

AGE-RELATED DISEASES

GENE THERAPY
   • Gene Therapy Strategies Targeting Aging-Related Diseases

AGE-RELATED MACULAR DEGENERATION

THERAPY
   • Adenovirus-mediated delivery of Factor H
   •gene therapies for age-related macular degeneration

AGING

GENE THERAPY
   • Anti-Aging Gene Therapy
   • clinical trials with stem cells to slow or reverse normal aging processes

ADRENAL DISEASES

CELL- and GENE-THERAPY
   • Novel treatments for adrenal diseases: Cell- and gene therapy-based approaches

ADRENOLEUKODYSTROPHY

UNORTHODOX TREATMENT
  ◊ Oleic e erucic acids

GENE THERAPY
  ◊ Hematopoietic Stem-Cell Gene Therapy
  ◊ Gene editing via HITI for adrenoleukodystrophy treatment

AMYOTROPHIC LATERAL SCLEROSIS

GENE THERAPY
  ◊ Gene therapy for ALS
  ◊ progress Gene therapy for ALS

ANTI-CANCER THERAPY

ROLE of lncRNAs
  ◊ Molecular Mechanisms of lncRNAs in the Dependent Regulation of Cancer and Their Potential Therapeutic Use

ANTI-ANGIOGEENIC THERAPY

ANGIOGENESIS-DEPENDENT DISEASES
  ◊ Therapeutic targeting of angiogenesis molecular pathways

ANTI-AAV ANTIBODIES by GENE THERAPY

BINDING AND NEUTRALIZING
  ◊Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy

ANTISENSE OLIGONUCLEOTIDE-BASED THERAPY

NEUROMUSCULAR DISEASE
  ◊ Therapу DMD and SMA

ALKAPTONURIA

GENE THERAPY
  ◊ Control of alkaptonuria with nitisinone and gene therapy

ALLERGY AND ASTHMA

  ◊ Chimeric antigen receptor-T cell therapy of allergy and asthma
  ◊ Airway epithelial-targeted nanoparticles for asthma therapy

ALZHEIMER'S DISEASE

THERAPEUTIC APPROCHES
  ◊ Diagnostic and Therapeutic approches
  ◊ Neuroprotective effects of a triple GLP-1/GIP/glucagon receptor in mouse model
  ◊ Aggregated apoE with antibodies inhibits amyloid accumulation
  ◊ Clearance of Amyloid Beta and Tau in Alzheimer’s Disease
  ◊ PPAR-δ and erucic acid in multiple sclerosis and Alzheimer's Disease.
  ◊ Stem cell therapy in Alzheimer’s disease
  ◊ Advances, insights, and prospects of gene therapy for AlzheimerТs disease

GENETHERAPY
  ◊ Gene therapy-mediated enhancement of protective protein expression for the treatment of Alzheimer’s disease AMYLOID LIGHT-CHAIN (AL)

DRUG THERAPY
  ◊ Approaches

β-AMILOID PROTEIN
  ◊ Nonsteroidal anti-inflammatory drugs
  ◊ Processing by BACE1,Alzheimer's β-secretase

  ◊ Small-molecule inhibitors of Aβ
  ◊ Therapeutic approches

DENDRITIC SPINE LOSS
  ◊ Phenylbutyrate rescues

DIAGNOSTIC AND THERAPY
  ◊ Approches
  ◊ Nornicotine
  ◊ Tau immunotherapy

LATE ONSET ALZHEIMER DISEASE
  ◊ Retinoid Receptors

NERVE CELL DEATH
  ◊ Cathepsin B

ALLERGIC RHINITIS

NEW TARGETS
  ◊ Allergen-specific IgE

ANALGETICS

BRADYKININ RECEPTOR LIGANDS
  ◊ Therapeutic perspectives

ANTI-FIBROTIC MOLECULES

PROTEIN THERAPY
  ◊Organ-specific, anti-fibrotic molecule by molecular engineering of the extracellular matrix protein, decorin

ANTISENSE OLIGONUCLEOTIDE-BASED THERAPY

NEUROMUSCULAR DISEASE
  ◊ Therapo DMD and SMA

AMYOTROPHIC LATERAL SCLEROSIS

THERAPEVTIC APPROACHES
  ◊ Drugs and Antisense oligonucleotides
  ◊ Approaches

GENEN THERAPY
  ◊ Gene therapy for ALS:

ANTIBIOTICS

NEW
  ◊ Daptomycin

ANTIDEPRESSANT

NEW
  ◊ Leptin

DISEASE LU GERIG
  ◊ Therapeutic approches

ANGELMAN SYNDROME

ARTIFICIAL TRANSCRIPTION FACTOR
  ◊ Restores Widespread Ube3a Expression in Mouse Brain

ANGIOGENESIS IN DISEASE

PRO- OR ANTI-ANGIOGENESIS TREATMENTS
  ◊ Targetibg haematopoieic cells

ANNOTATED COMPOUND LIBRARY

SMALL MOLECULES
  ◊ Mimetics

ANTIPYRETIC DRUGS

IMMUNE-INDUCED PYRESIS
  ◊ Microsomal prostaglandin E synthase-1

ANXIOLYTIC DRUGS

FREE OF SEDATIVE SIDE EFFECTS
  ◊ Antibiotic quinolone

APERT SYNDROME

POTENTIAL GENETIC THERAPY
  ◊ Insights and future directions of genetic therapy for Apert syndrome

APLASTIC ANEMIA

TRANSPLANTATION MARROW
  ◊ Alternative Donor Transplantation with High-Dose Post-Transplantation Cyclophosphamide

iPS CELLS
  ◊ Disease modelling using iPS cell-derived hematopoietic stem progenitor cells

APOPTOSIS

ULTRAVIOLET RADIATION-INDUCED
  ◊ Interleukin-12

APTAMER-BASED TARGETED THERAPY

APTAMER-BASED TARGETED THERAPY
  ◊ Aptamer-based targeted therapy

AROMATIC L-AMINO ACID DECARBOXYLASE DEFICIENCY

GENE THERAPY
  ◊ Gene therapy improves motor and mental function deficiency

ASTHMA

THERAPEVTIC EFFECTS
  ◊ BPIFA1/SPLUNC1
  ◊ Oxaprozin
  ◊ Hepatitis A affects asthma
  ◊ serevent

INHIBITION OF MUCUS HYPERSECRETION
  ◊ MARCKS-related peptide

FRIEDREICH'S ATAXIA

THERAPEVTIC EFFECTS
  ◊ Frataxin
  ◊ Hematopoietic stem and progenitor cells ameliorates deficits in a mouse model of Friedreich's ataxia

ATAZANAVIR SULPHATE

THERAPEVTIC EFFECTS
  ◊ HIV therapy

ATHEROGENIC DYSLIPIDEMIA

CRISPR-Cas9 GENOME EDITING
  ◊ CRISPR-Cas9 Genome Editing for Treatment of Atherogenic Dyslipidemia

ATRIAL FIBRILLATION

THERAPEVTIC EFFECTS
  ◊ Factor Xa inhibitors

AUDITORY NEUROPATHY SPECTRUM DISORDERS (ANSD)

GENE THERAPIES
  ◊ Current Advances in Gene Therapies of Genetic Auditory Neuropathy Spectrum Disorder

AUTISM SPECTRUM DISORDERS (ASDs)

CEREBELLAR DYSFUNCTION IN PURKINJE CELL
  ◊ Strategies therapy

DRUG THERAPY
  ◊ Histone deacetylase (HDAC) inhibition

  ◊ Translating precision medicine for autism spectrum disorder

GENE THERAPY
  ◊ Autism spectrum disorder: prospects for treatment using gene therapy

AUTOSOMAL DOMINANT DISORDERS

GENE THERAPY
  ◊ CRISPR/Cas therapeutic strategies for autosomal dominant disorders

AUTOIMMUNE DISEASES

BIOSIMILAR GENE THERAPY
  ◊ Investigational Assessment of Secukinumab Gene Therapy

GENOME EDITING
  ◊ Genome Editing Using CRISPR-Cas9 by Autoimmune Diseases

AUTOSOMAL DOMINANT RETINITIS PIGMENTOSA

GENE THERAPY
  ◊ Gene Therapy for Rhodopsin-associated Autosomal Dominant Retinitis Pigmentosa

AUTOSOMAL-DOMINANT CENTRONUCLEAR MYOPATHY (AD-CNM)

SILENCING THERAPY
  ◊ Allele-specific silencing therapy for Dynamin 2-related dominant centronuclear myopathy