JP6811857B2 - B型肝炎ウイルスゲノムの認識配列に特異的な遺伝子操作メガヌクレアーゼ - Google Patents
B型肝炎ウイルスゲノムの認識配列に特異的な遺伝子操作メガヌクレアーゼ Download PDFInfo
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Description
本願は、EFS−Webを介してASCIIのフォーマットで提出された配列表を含み、その全体は参照により本明細書に組み入れられる。2017年10月13日に作成された上記のASCIIのコピーは、P109070018WO00−SEQ−MJT.txtという名前で、サイズは169,011バイトである。
配列番号1は、緑藻クラミドモナスからの野生型I−CreIメガヌクレアーゼのアミノ酸配列を示す。
Claims (15)
- 配列番号16からなる認識配列と結合して切断する、遺伝子操作されたメガヌクレアーゼであって、
第1のサブユニット及び第2のサブユニットを含み、
前記第1のサブユニットが前記認識配列の第1の認識した半分の部位に結合し、
前記第2のサブユニットは、前記認識配列の第2の認識した半分の部位に結合し、
配列番号33と少なくとも96%の配列同一性を有するアミノ酸配列を含む、遺伝子操作されたメガヌクレアーゼ。 - 前記第1のサブユニットが、配列番号33の残基198〜344に対して少なくとも96%の配列同一性を有するアミノ酸配列を含み、前記第2のサブユニットが、配列番号33の残基7〜153に対して少なくとも96%の配列同一性を有するアミノ酸配列を含む、請求項1に記載の遺伝子操作されたメガヌクレアーゼ。
- 前記第1のサブユニットが、配列番号33の残基198〜344を含む、請求項1又は2に記載の遺伝子操作されたメガヌクレアーゼ。
- 前記第2のサブユニットが、配列番号33の残基7〜153を含む、請求項1〜3のいずれか1項に記載の遺伝子操作されたメガヌクレアーゼ。
- 配列番号33のアミノ酸配列を含む、請求項1〜4のいずれか1項に記載の遺伝子操作されたメガヌクレアーゼ。
- 請求項1〜5のいずれか1項に記載の遺伝子操作されたメガヌクレアーゼをコードする核酸配列を含む、ポリヌクレオチド。
- 前記ポリヌクレオチドがmRNAである、請求項6に記載のポリヌクレオチド。
- 請求項1〜5のいずれか1項に記載の遺伝子操作されたメガヌクレアーゼをコードする核酸配列を含む、組換えDNA構築物。
- 請求項1〜5のいずれか1項に記載の遺伝子操作されたメガヌクレアーゼをコードする核酸配列を含む、ウイルスベクター。
- 前記ウイルスベクターが組換えアデノ関連ウイルス(AAV)ベクターである、請求項9に記載のウイルスベクター。
- B型肝炎ウイルス(HBV)又はHBVに起因する肝細胞がんを有する対象を治療するための医薬組成物であって、医薬的に許容される担体及び請求項1〜5のいずれか1項に記載の遺伝子操作されたメガヌクレアーゼをコードする核酸を含む、医薬組成物。
- 前記遺伝子操作されたメガヌクレアーゼをコードする前記核酸がmRNAである、請求項11に記載の医薬組成物。
- 前記mRNAは、脂質ナノ粒子内に封入されている、請求項12に記載の医薬組成物。
- 前記遺伝子操作されたメガヌクレアーゼをコードする前記核酸を含むウイルスベクターを含む、請求項11に記載の医薬組成物。
- 前記ウイルスベクターが組換えAAVベクターである、請求項14に記載の医薬組成物。
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JP2021061841A (ja) * | 2016-10-14 | 2021-04-22 | プレシジョン バイオサイエンシズ,インク. | B型肝炎ウイルスゲノムの認識配列に特異的な遺伝子操作メガヌクレアーゼ |
US11274285B2 (en) | 2016-10-14 | 2022-03-15 | Precision Biosciences, Inc. | Engineered meganucleases specific for recognition sequences in the Hepatitis B virus genome |
US11142750B2 (en) | 2018-04-12 | 2021-10-12 | Precision Biosciences, Inc. | Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome |
US11788077B2 (en) | 2018-04-12 | 2023-10-17 | Precision Biosciences, Inc. | Polynucleotides encoding optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome |
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