Central nervous system (CNS) diseases include diverse group of conditions in which the brain loses its normal functioning, thereby reducing the capacity to operate day to day life activities. These can occur due to several reasons such as hereditary, others are caused by injury, and few are caused by infections. The most difficultly in providing treatments for CNS diseases is allowing adequate amount of blood–brain barrier (BBB) penetration. Several Cell and Gene Therapies (CGTs) are in various stages of clinical development for targeting the most destructive CNS disorders, including spinal muscular atrophy (SMA), Parkinson's disease, Alzheimer's disease (AD), multiple sclerosis (MS), Huntington’s disease (HD), spinal cord injury (SCI), and amyotrophic lateral sclerosis (ALS).
Cell therapy for CNS has advanced to new level of clinical applications. Several clinical trials are investigating potential cell therapy for various CNS disorders such as stroke, traumatic brain injury, Parkinson's disease and other neurological diseases. The transplanted cells can serve as a member of the newly formed network in the host tissue and are capable of secreting different trophic factors as well as neuroprotective & neurorestorative properties. Gene transfer technologies are responsible for improving illness conditions in patients. Therapeutic genes can effectively protect against the neurodegenerative disease.
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