RU2016104674A - Устройство модификации эритроцита с механизмом направленного транспорта лекарственного средства для функций генной терапии crispr/cas9 - Google Patents
Устройство модификации эритроцита с механизмом направленного транспорта лекарственного средства для функций генной терапии crispr/cas9 Download PDFInfo
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- 210000003743 erythrocyte Anatomy 0.000 title claims 21
- 108091033409 CRISPR Proteins 0.000 title claims 6
- 238000010354 CRISPR gene editing Methods 0.000 title claims 2
- 238000001415 gene therapy Methods 0.000 title 1
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- 238000012986 modification Methods 0.000 title 1
- 230000007723 transport mechanism Effects 0.000 title 1
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- 108010010803 Gelatin Proteins 0.000 claims 4
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- 239000008273 gelatin Substances 0.000 claims 4
- 235000019322 gelatine Nutrition 0.000 claims 4
- 235000011852 gelatine desserts Nutrition 0.000 claims 4
- 239000000243 solution Substances 0.000 claims 4
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims 3
- 239000003814 drug Substances 0.000 claims 3
- 229940079593 drug Drugs 0.000 claims 3
- 108700011259 MicroRNAs Proteins 0.000 claims 2
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- 238000005406 washing Methods 0.000 claims 2
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- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 238000013270 controlled release Methods 0.000 claims 1
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- 239000007924 injection Substances 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 238000003780 insertion Methods 0.000 claims 1
- 230000037431 insertion Effects 0.000 claims 1
- 239000006194 liquid suspension Substances 0.000 claims 1
- 239000002679 microRNA Substances 0.000 claims 1
- 239000002504 physiological saline solution Substances 0.000 claims 1
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Claims (9)
1. Лекарственное средство в форме жидкой суспензии отмытых модифицированных эритроцитов, предназначенной для инъекционного применения, с замедленным регулируемым высвобождением в организме лекарственного препарата и обеспечивающих эффективную направленность лекарства, отличающееся тем, что производят проведение трехкратной отмывки эритроцитов в физиологическом растворе, ресуспензирование эритроцитов в изотоническом растворе низкой ионной силы нанокапсулированного желатина, содержащего в середине микроРНК (sgRNAs), белок Cas9 и последовательность ДНК для внедрения, суккуссию суспензии эритроцитов в закрытой пробирке с температурным и тензометрическим датчиком посредством ударов о твердую эластичную поверхность, добавление антител определенной специфичности, инкубацию пробирки в термостате, проведение трехкратной отмывки эритроцитов в физиологическом растворе, ресуспензирование модифицированных эритроцитов в изотоническом растворе натрия хлорида.
2. Суспензия отмытых модифицированных эритроцитов по п. 1, отличающаяся тем, что в центре нанокапсулированного желатина заключаеться упаковываная в вирус CRISPR/Cas9 - кассета, включающая молекулы микроРНК (sgRNAs), белок Cas9 и последовательность ДНК для внедрения.
3. Суспензия отмытых модифицированных эритроцитов по п. 1, отличающаяся тем, что в пробирку с эритроцитарной массой добавляют 8% раствор нанокапсулированного желатина в изотоническом растворе низкой ионной силы Low Ionic Strength Solution (Liss) при температуре 36,3-36,9°C, предпочтительно 36,6°C, рН раствора должно быть равным 7,00-7,40, предпочтительно рН 7,35.
4. Суспензия отмытых модифицированных эритроцитов по п. 1, отличающаяся тем, что пробирку заполнят по объему на 1/2-2/3 суспензией эритроцитов.
5. Суспензия отмытых модифицированных эритроцитов по п. 1, отличающаяся тем, что производят суккуссию суспензии посредством ударов о твердую эластичную поверхность при температуре 36,3-36,9°С, предпочтительно 36,6°С, величину повышения давления при гидравлическом ударе о дно сосуда суспензии эритроцитов контролируют тензометрическим датчиком, работающим в комплекте со шлейфовым осциллографом, давление составляет 2,2-2,4 кПа, предпочтительно 2.3 кПа, время суккуссии суспензии эритроцитов от 30 секунд до 2 минут, предпочтительно 30 секунд.
6. Суспензия отмытых модифицированных эритроцитов по п. 1, отличающаяся тем, что в изотонический раствор низкой ионной силы 8% нанокапсулированного желатина добавляют антитела определенной специфичности, инкубируют в термостате от 15 минут до 1 часа, предпочтительно 30 минут при температуре 36,6°С.
7. Суспензия отмытых модифицированных эритроцитов по п. 1, отличающаяся тем, что отмывают эритроциты по три раза: центрифугируют в физиологическом растворе в течение 10 минут при 3000 об/мин, при температуре 36,3-36,9°С, предпочтительно 36,6°С, рН раствора должно быть равным 7,00-7,40, предпочтительно рН 7,35.
8. Суспензия отмытых модифицированных эритроцитов по п. 1, отличающаяся тем, что инфузия пациенту производится при температуре суспензии 36,6°С.
9. Суспензия отмытых модифицированных эритроцитов по любому из пп. 1-6, отличающаяся тем, что применяют аутологичные эритроциты.
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