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Gene Therapy in ALS and SMA: Advances, Challenges and Perspectives

6 Jan 2023

Abstract excerpt

Gene therapy is defined as the administration of genetic material to modify, manipulate gene expression or alter the properties of living cells for therapeutic purposes. Recent advances and improvements in this field have led to many breakthroughs in the treatment of various diseases. As a result, there has been an increasing interest in the use of these therapies to treat motor neuron diseases (MNDs), for which...

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PMID: 36674643
DOI: 10.3390/ijms24021130

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