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May 5, 1993

Gene Therapy for Brain Tumors in Trials, Correction of Inherited Disorders a Hope

JAMA. 1993;269(17):2181-2182. doi:10.1001/jama.1993.03500170011002

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CLINICIANS and laboratory scientists alike have coveted the ability to genetically alter brain cells. Were this manipulation possible, brain cancer conceivably could be cured. So could the multitude of other acquired and inherited diseases that involve the central nervous system.

From a basic science standpoint, researchers could pry open the mysteries of neural development and other enigmas by studying the function and regulation of cloned genes in animal models.

For many years, however, the brain has proven intractable to the embryonic science of gene transfer. Because brain cells do not proliferate, the early protocols that required DNA replication for the delivery of genetic material were ineffective. Moreover, the blood-brain barrier rendered the brain impenetrable to systemic-based therapies.

But scientists are learning to overcome these obstacles and in some cases even use them to their advantage. Eight patients have now been treated in the first gene therapy trial for malignant brain