MACULAR DEGENERATION

IN CANINE MODEL
  ◊ BEST1 gene therapy corrects a diffuse retina-wide microdetachment

NEOVASCULAR AGE-RELATED MACULAR DEGENERATION
  ◊Multiple gene therapy as a tool for regulating the expression of molecules involved in neovascular age-related macular degeneration

MAPLE SYRUP URINE DISEASE

GENE THERAPY
  ◊ Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice

AGE-RELATED MACULAR DEGENERATION
  ◊ therapy with embryonic stem cell-derived retinal pigment epithelium patch

MELANOMA

THERAPEUTIC STRATEGY
  ◊ TERT dependency as a therapeutic strategy for NRAS-mutant melanoma
  ◊ A TCF4-dependent gene regulatory network confers resistance to immunotherapy in melanoma

MALARIA

VACCINE
  ◊ Trials

MALFORMATIONS

DRUGS
  ◊ Antithyroid Drugs

MANGANESE METABOLISM

THERAPEUTIC APPROACHES
  ◊ Genetic Disorders of Manganese Metabolism

alpha-MANNOSIDOSIS

GENE THERAPY
  ◊ Correction of human alpha-mannosidosis by intravascular gene therapy

MARFAN Syndrome

DRUGS
  ◊ Losartan

MEMORY DEFICITS

GENE THERAPY
  ◊ αKlotho isoform protects against age-dependent memory deficits

MELANOMA

TARGETED THERAPY
  ◊ Advances
  ◊ Efficacious schedules of MEK1/2-CDK4/6 targeting

MICROGLIAPATY

GENE THERAPY
  ◊ Hematopoietic stem cell gene therapy to halt neurodegeneration

MICROVILLUS INCLUSION DISEASE

THERAPY
  ◊ The challenge of personalized cell biology

miRNAs THERAPEUTICS

PROGRESS
  ◊ Advances
  ◊ microRNA tools in molecular diagnostics and therapeutics

MIGRAINE

POLYTHERAPY
  ◊ Advances

MITOCHONDRIAL DISEASES

GENE THERAPY
  ◊ gene therapy for mitochondrial diseases
  ◊ Mitochondrial DNA heteroplasmy and targeted nucleasebased therapeutic approaches
  ◊ Strategies for mitochondrial gene editing
  ◊ Gene therapy for mitochondrial disorders
  ◊ Gene therapy for mitochondrial disorders

MITOCHONDRIAL NEUROGASTROINTESTINAL ENCEPHALOMYOPATHY

GENE THERAPY
  ◊ Preclinical Assessment of a Gene Editing Approach in a Mouse Model of MNGIE

mRNA-BASED THERAPEUTICS

GENE THERAPY
  ◊ mRNA-based therapeutics: powerful and versatile tools to combat diseases

MODULATORS OF THE HEDGEHOG-GLI SIGNALING PATHWAY

THERAPEUTIC POTENTIAL
  ◊ : Current status

MUCOPOLYSACCHARIDOSES

GENE THERAPY
  ◊ Gene therapies for mucopolysaccharidoses

MOLECULAR DYSMORPHOLOGY

GENETIC MEDICINE
  ◊ gene therapy

MULTIPLE MYELOMA

THERAPY PROTEASOME INHIBITORS
  ◊ Bortezomib

MULTIPLE SCLEROSIS

INHIBITORS OF INFLAMATION
  ◊ Tissue factor and protein C inhibitor
  ◊ Neural stem cells to suppress chronic neuroinflammation

REVERSE DISEASE
  ◊ Gene Therapy-Induced Antigen-Specific Tregs

MUSCLE ATROPHY

PREVENT ATROPHY
  ◊ Serum/glucocorticoid-induced kinase 1

MUSCULAR DYSTROPHY

DRUG THERAPY
  ◊ Tamoxifen and raloxifene

THERAPEUTICS
  ◊ Gene-editing, immunological and iPSCs based therapeutics for muscular dystrophy
  ◊ Bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders

MUTAGENESIS

MEASURING
  ◊ Measuring Mutagenesis with Precision Genome Editing

MYOPATHIES RyR1-RELATED

GENETHERAPY
  ◊ Gene therapies for RyR1-related myopathies

MYOTONIC DYSTROPHY TYPE 1 and 2

GENETHERAPY
  ◊ Elimination of Toxic Microsatellite Repeat Expansion RNA

REVERSAL OF MOLECULAR PATHOLOGY
  ◊ RNA-targeting Cas9

MYOCARDITIS

GENE THERAPY VECTOR-RELATED
  ◊ Gene therapy vector-related myocarditis Cells

MYOCARDIAL INFARCTION

CELL THERAPY TO REGENERATE INFARCTED MYOCARDIUM
  ◊ Cartilage-Derived Stromal Cells

CYTOCHROME P450 INHIBITORS
  ◊ Chloramphenicol

MYOTONIC DYSTROPHY TYPE i (DM1)