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Challenges of CRISPR/Cas-Based Cell Therapy for Type 1 Diabetes: How Not to Engineer a "Trojan Horse".
Karpov, Dmitry S; Sosnovtseva, Anastasiia O; Pylina, Svetlana V; Bastrich, Asya N; Petrova, Darya A; Kovalev, Maxim A; Shuvalova, Anastasija I; Eremkina, Anna K; Mokrysheva, Natalia G.
Afiliación
  • Karpov DS; Engelhardt Institute of Molecular Biology, Russian Academy of Sciences, 119991 Moscow, Russia.
  • Sosnovtseva AO; Engelhardt Institute of Molecular Biology, Russian Academy of Sciences, 119991 Moscow, Russia.
  • Pylina SV; Endocrinology Research Centre, 115478 Moscow, Russia.
  • Bastrich AN; Endocrinology Research Centre, 115478 Moscow, Russia.
  • Petrova DA; Endocrinology Research Centre, 115478 Moscow, Russia.
  • Kovalev MA; Engelhardt Institute of Molecular Biology, Russian Academy of Sciences, 119991 Moscow, Russia.
  • Shuvalova AI; Engelhardt Institute of Molecular Biology, Russian Academy of Sciences, 119991 Moscow, Russia.
  • Eremkina AK; Endocrinology Research Centre, 115478 Moscow, Russia.
  • Mokrysheva NG; Endocrinology Research Centre, 115478 Moscow, Russia.
Int J Mol Sci ; 24(24)2023 Dec 10.
Article en En | MEDLINE | ID: mdl-38139149
ABSTRACT
Type 1 diabetes mellitus (T1D) is an autoimmune disease caused by the destruction of insulin-producing ß-cells in the pancreas by cytotoxic T-cells. To date, there are no drugs that can prevent the development of T1D. Insulin replacement therapy is the standard care for patients with T1D. This treatment is life-saving, but is expensive, can lead to acute and long-term complications, and results in reduced overall life expectancy. This has stimulated the research and development of alternative treatments for T1D. In this review, we consider potential therapies for T1D using cellular regenerative medicine approaches with a focus on CRISPR/Cas-engineered cellular products. However, CRISPR/Cas as a genome editing tool has several drawbacks that should be considered for safe and efficient cell engineering. In addition, cellular engineering approaches themselves pose a hidden threat. The purpose of this review is to critically discuss novel strategies for the treatment of T1D using genome editing technology. A well-designed approach to ß-cell derivation using CRISPR/Cas-based genome editing technology will significantly reduce the risk of incorrectly engineered cell products that could behave as a "Trojan horse".
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Diabetes Mellitus Tipo 1 Límite: Humans Idioma: En Revista: Int J Mol Sci Año: 2023 Tipo del documento: Article País de afiliación: Rusia

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Diabetes Mellitus Tipo 1 Límite: Humans Idioma: En Revista: Int J Mol Sci Año: 2023 Tipo del documento: Article País de afiliación: Rusia