LEBER CONGENITAL AMAUROSIS

GENE THERAPY
  ◊ Recombinant adeno-associated viruses (rAAVs)
  ◊ Genetic treatment for blindness
  ◊ Leber Congenital Amaurosis Clinical Trials

LEUKEMIA

CAR-T THERAPY
  ◊ Leukemia: CAR-T gene therapy
  ◊ CRISPR-directed gene editing as a therapeutic strategy for leukemia and beyond
  ◊A toolmaker’s perspective on CRISPR-directed gene editing as a therapeutic strategy for leukemia and beyond

LIMB GIRDLE MUSCULAR DYSTROPHY 2B

GENE THERAPY
  ◊ Secreted acid sphingomyelinase as a potential gene therapy for limb girdle muscular dystrophy 2B

LIMBAL STEM CELL DEFICIENCY (LSCD)

THERAPY
  ◊ The Multifold Etiologies of Limbal Stem Cell Deficiency: Treatment Options for Limbal Stem Cell Deficiency

LIPIDEMIAS

GENE EDITING
  ◊ Gene editing for dyslipidemias: New tools to “cut” lipids

LIPID NANOPARTICLES

CRISPR–Cas GENE EDITING
  ◊ Membrane-destabilizing ionizable phospholipids and CRISPR–Cas gene editing

LIVER DISEASES

THERAPY
  ◊ Human antigen R: A potential therapeutic target for liver diseases
  ◊Reversal of liver failure using a bioartificial liver device implanted with clinical-grade human-induced hepatocytes

GENE THERAPY
  ◊ Novel vectors and approaches for gene therapy in liver diseases
  ◊ In vivo Gene Therapy to the Liver and Nervous System: Promises and Challenges
  ◊ Liver-directed gene therapy for inherited metabolic diseases

LONG-TERM EXPRESSION OF BIOTHERAPEUTICS

GENE THERAPY
  ◊ gene therapy to achieve long-term continuous or controllable expression of biotherapeutics

LUNG DISEASES

GENE THERAPY
  ◊ New Directions in Pulmonary Gene Therapy

LIVER FIBROSIS

THERAPY
  ◊ Overexpression of Mitofusin-2 in Hepatic Stellate Cells Ameliorates Liver Fibrosis

LUNG ADENOCARCINOMA

IMMUNE CHECKPOINT BLOCKADE mRNA
  ◊ Lung adenocarcinoma immune checkpoint blockade mRNA expression B cells

LUNG DISEASES

GENE EDITING
  ◊ In Utero Gene Editing for Inherited Lung Diseases

LUNG FIBROSIS

DRUG THERAPY
  ◊ Metformin reverses established lung fibrosis in a bleomycin model.

LUNG TRANSPLANTATION

GENE THERAPY TRANSPLANT
  ◊ Creating superior lungs for transplantation with next-generation gene therapy during ex vivo lung perfusion

LYMPHEDEMA

THERAPY
  ◊ Immediate lymphatic reconstruction for the prevention of breast cancer-related lymphedema

LYMPHATIC MALFORMATIONS

THERAPY
  ◊ Genetic and Molecular Determinants of Lymphatic Malformations: Potential Targets for Therapy

LYSOSOMAL STORAGE DISORDER

CELL THERAPY
  ◊ Intracerebroventricular delivery of hematopoietic progenitors results in rapid and robust engraftment of microglia-like cells

GENETHERAPY
  ◊ Effects of gene therapy on cardiovascular symptoms
  ◊ Molecular Pathology and Developing Therapies for Lysosomal Storage Disorders
  ◊ Gene therapy for lysosomal storage diseases: Current clinical trial prospects