There have been many ups and downs since the introduction of gene therapy as a
therapeutic modality for diseases. However, the journey of gene therapy has reached a …

The first cancer-targeted microRNA (miRNA) drug—MRX34, a liposome-based miR-34
mimic—entered Phase I clinical trials in patients with advanced hepatocellular carcinoma in …

Abstract Adeno-associated virus (AAV)-based vectors are promising tools for gene
therapeutic applications, in part because AAVs are nonpathogenic viruses, and vectors …

Background: In 1989, the concept of human gene therapies has emerged with the first
approved human gene therapy trial of Rosenberg et al. Gene therapies are considered as …

Oncolytic viruses (OV) are an attractive prospect due to their dual attack mechanism of direct
cell lysis and potentiation of an antitumor immune response. Various oncolytic viral vectors …

Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, is
characterized by the deterioration of alpha motor neurons in the brainstem and spinal cord …

The field of genome engineering has created new possibilities for gene therapy, including
improved animal models of disease, engineered cell therapies, and in vivo gene repair. The …

Mucopolysaccharidoses (MPS) are a group of lysosomal storage disorders (LSDs) caused
by a deficiency of lysosomal enzymes, leading to a wide range of various clinical symptoms …

For many neuromuscular disorders, including Duchenne muscular dystrophy, spinal
muscular atrophy and myotonic dystrophy, the genetic causes are well known. Gene therapy …

Abstract The Neuronal Ceroid Lipofuscinoses (NCLs) are a family of autosomal recessive
neurodegenerative disorders that annually affect 1: 100,000 live births worldwide. This …