?????????协??_365体育比分
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????????2022/8/16 23:41:20
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??????????????????Precision BioSciences???????????27??????????????????????????????(DMD)?????????????????芯??????????????????????
?????????????(DMD)?????????????????(Dys)?????????????????????????????????????????X?????????????????????????????????携?????????????DMD??????械????????1/6000-1/3500?????寤??????15/100000-20/100000?????????????????4???????????????????卸?????????????????????12?????????????????械???校?????????20?????????????????????????????????
??????????DMD???????????????????????????????
???????????????DMD??????????????????????????????????????????????????胁???????????????????????????DMD????????????Sarepta Therapeutics???????????????FDA??2016??2019????????校??????Eteplirsen(Exondys 51)??Golodirse(Vyondys 53)?????????45/51/53??????????????????????????蟹????小?????????????????效????????????????????????????????????????????渭??????
??????????????????????DMD????????????????胁???????????DMD??????谐???????????????????????????????????????????????????????璺???????????????????AAV?????榈?????????????????????????????2600kb????????AAV???????????????????????宸?????谩??????????????????????????????????????????????些???????薪????????AAV???????澹??????????????????梅??????????????????????
??????????????????????绮??DMD????????????????2016??????????????????Bamboo Therapeutics????DMD??????????蟹????????????5?路????????蟹????????????????????Sarepta Therapeutics??????芯????DMD????????SRP-9001?????????????????
????Grand View Research??????????2023?????DMD?????谐????????41.1?????????????41.3%??????????????????
????????????????????????????DMD???????????????
???????????????????芯??????
1????????Precision BioSciences
???????????Precision BioSciences?????????协?椋?????????Precision??ARCUS????????????芯?????????????????????????????????????????斜?????DMD??????芯???????????Precision??????????????芯????泄?????芯?????(IND)?????????????????????????????????????
?????ARCUS??Precision????????????懈??????????????????????????????????????械?DNA?懈????????????????(????)?????(?贸?)???????????????????DNA??????I-CreI???????????????????????????槌?????????????????????????????校?????????DNA???懈??????????懈?????????????????????????????????????????????????位??
????????ARCUS?????????????????????DMD?????????????????????????????????????????????(adRP)???????????????(FH)?????????????????????????????(ATTR)??????ARCUS????DMD?????????????????????45-55??????????????????????
??魏???????????????????????Precision??ARCUS????????????????????????????????????????????????????效??????蔚?????????????????????????????????????????????
2????????Bamboo Therapeutics
2020??11????????????????????????DMD???芯?????????????PF-06939926?????????FDA????????????????
??????????PF-06939926 Ib????????????????????9???????DMD?泻???????????????PF-06939926?????????????????????????????????????????????????12?????
PF-06939926???????2018????????Ib????????????????????????????????芯????????芯?????????????????芯???????????????????????PF-06939926????AAV9???澹??????????姹??????????????????????????????????????????????AAV9?????邪?????????????????????????????濉
3??Sarepta??????
Sarepta Therapeutics??????????????????????????????DMD????????????SRP-9001??2020??9???Sarepta??????SRP-9001???芯?????????????4????????????4-7????2??????????????????????????SRP-9001??2??NSAA??????????????????????2???????????谢????SRP-9001???????????谩?
SRP-9001???????????????????????????????????????????????????????????????????????????DMD??????????????????????????(Dystrophin)?????未???SRP-9001???写???????效?????????2019??12?????????Sarepta????????28.5???????????协?椋?????SRP-9001???????????????????????Sarepta????SRP-9001?????????????????????????SRP-9001???????
2020??7???SRP-9001???????FDA??????????DMD??????????????SRP-9001????FDA???????????????????SRP-9001???????????????????????????????
???DMD??????谢????????????????????????????Sarepta????????????械?????芯???????????????????????????????
???????????????纾??
(2020/12/5 10:07:07      ???31724??)

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