The other day, I attended a seminar on gene therapy.
I got the impression that Japan is still lagging behind, with research and clinical trials progressing in Europe and the United States.
Is gene therapy possible for TANGO2? Are there plans to do so?
Please let me know if the topic has already been discussed and there are any materials available.
@linaghaloul @jmeisner @Cmiyake @seemal @samuel_mackenzie @Ryan_Carrier @mousumibose @cosmo @HdeCalbiac @Majoarredondo @nicolasabreumd
Genetic therapies are being used to treat a wide range of conditions from rare genetic disorders to cancer. For more information about gene therapy, consider https://patienteducation.asgct.org/
Whether or not gene therapy is safe or effective for TANGO2 deficiency disorder is unknown. One potential strategy is gene addition, since TANGO2 deficiency disorder typically results from not having enough TANGO2 (Different Approaches | ASGCT - American Society of Gene & Cell Therapy |).
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