Genes & CellsGenes & CellsГены и КлеткиGenes and Cells2313-18292500-2562Human Stem Cells Institute12237210.23868/gc122372ReviewsНаучные обзорыReview ArticleGene and stem-cell therapy for neurodegenerative diseasesГенная и клеточная терапия нейродегенеративных заболеванийIslamovR. R.ИсламовР. Р.
Russian Federation
islamru@yahoo.comRizvanovА. А.РизвановА. А.
Russian Federation
islamru@yahoo.comGusevaD. S.ГусеваД. С.
Russian Federation
islamru@yahoo.comKiasovA. P.КиясовА. П.
Russian Federation
islamru@yahoo.comKazan State Medical UniversityКазанский государственный медицинский университетKazan State UniversityКазанский государственный университетStem-cell bank of Kazan State Medical UniversityБанк стволовых клеток Казанского государственного медицинского университета150920072329371601202316012023Copyright ©; 2007, Eco-VectorCopyright ©; 2007, Эко-Вектор2007Eco-VectorЭко-Векторhttps://genescells.ru/2313-1829/article/view/122372

Currently there are no available effective therapies for treating neurodegenerative diseases. In animal models, the loss of neurons, caused by mutations in known genes, can be alleviated by genetically modifying target cells to increase their regeneration and viability, or by replacing dead neurons with new healthy neural cells by neurotransplantation of stem or progenitor cells, differentiated in neural pathway. Here we summarized results for gene therapy using antisense oligonucleotides, siRNA, and viral vectors. Critically reviewed advantages and disadvantages of neurotransplantation of embryonic and different adult stem cells. To date we found no reports of using genetically modified stem cells from umbilical cord blood for cell therapy of neurodegenerative diseases. We state a hypothesis, that stem cells from umbilical cord blood, genetically modified by transfection with plasmid vector, simultaneously expressing neural cell adhesion molecule L1 and vascular endothelial growth factor [VEGF), could have a significantly enhanced therapeutic effect in transgenic mice G93A, which serve as animal model for amyotrophic lateral sclerosis [ALS).

Эффективных методов лечения нейродегенеративных заболеваний на сегодняшний день не существует. В эксперименте потеря нейронов, вызванная мутациями известных генов, может быть остановлена путём введения в геном клетки-мишени терапевтического гена с целью повышения жизненной стойкости нейрона, или путём замены погибших нейронов на молодые здоровые нервные клетки путём нейротрансплантации стволовых клеток или их потомков, пре- дифференцированных в нейрональном направлении. В настоящем обзоре представлены модели нейродегенеративных заболеваний на животных и новые направления в терапии нейродегенерации разной этиологии, основанные на генетической модификации нейронов в очаге дегенерации, а также стволовых клеток перед нейротрансплантацией. Нами обобщены результаты по генной терапии с помощью антисмысловых олигонуклеотидов, РНК-интерференции и вирусных систем. Рассмотрены преимущества и недостатки нейротрансплантации эмбриональных и разных стволовых клеток взрослого организма. До настоящего времени в мировой научной литературе отсутствовали прямые указания на возможность применения генетически модифицированных клеток пуповинной крови для клеточной терапии нейродегенеративных заболеваний. На основании чего представлена гипотеза о том, что генетически модифицированные стволовые клетки пуповинной крови, трансфицированные плазмидным вектором, одновременно экспрессирующим клонированные нейрональную молекулу адгезии L1 и сосудистый эндотелиальный фактор роста [VEGF), значительно усилят терапевтический эффект стволовых клеток пуповинной крови у трансгенных G93A мышей с фенотипом бокового амиотрофического склероза.

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