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The location of integration of a gene therapy has been crucial for the safety and efficacy of the treatment to cure infants with X-linked severe combined immunodeficiency.
Drs Gottschalk and Krenciute discuss the difficulties of translating successes with CAR T-cell therapy in adults to the pediatric population; ongoing research with investigational products that could reduce T-cell exhaustion, burnout, and the time from bench to bedside; and expectations for the future of cellular therapy in oncology.
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Adding a molecular anchor to the key protein used to recognize cancer in cellular immunotherapies can make the treatments significantly more effective.
Ten children with an especially rare and hard-to-treat form of "bubble boy" disease are living normal lives after receiving a new gene therapy approach, researchers say.
T cells used in immunotherapy treatments can get exhausted and shut down by fighting cancer cells and tumors. Using a CRISPR-based edit on these cells’ genomes, researchers at UCSF and Gladstone Institutes have rendered the therapeutic cells more resilient against tumors.