CN108699542A - 用于治疗角膜营养不良的方法 - Google Patents

用于治疗角膜营养不良的方法 Download PDF

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Publication number
CN108699542A
CN108699542A CN201680077123.2A CN201680077123A CN108699542A CN 108699542 A CN108699542 A CN 108699542A CN 201680077123 A CN201680077123 A CN 201680077123A CN 108699542 A CN108699542 A CN 108699542A
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nucleic acid
lys
leu
corneal dystrophy
ile
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Chinese (zh)
Inventor
T.莫尔
A.内斯比特
G.李
S-Y.乔
L.德迪奥尼西奥
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Avellino Lab USA Inc
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Avellino Lab USA Inc
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CN201680077123.2A 2015-11-13 2016-11-14 用于治疗角膜营养不良的方法 Pending CN108699542A (zh)

Applications Claiming Priority (3)

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US201562255310P 2015-11-13 2015-11-13
US62/255,310 2015-11-13
PCT/US2016/061893 WO2017083852A1 (en) 2015-11-13 2016-11-14 Methods for the treatment of corneal dystrophies

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US (1) US11987809B2 (enExample)
EP (2) EP3374502B1 (enExample)
JP (2) JP6929865B2 (enExample)
KR (1) KR20180120670A (enExample)
CN (1) CN108699542A (enExample)
ES (1) ES2905558T3 (enExample)
WO (1) WO2017083852A1 (enExample)

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CN114245741A (zh) * 2019-03-28 2022-03-25 儿童医学中心公司 高功能性的制造的abcb5+间充质干细胞
CN114729365A (zh) * 2019-03-19 2022-07-08 布罗德研究所股份有限公司 用于编辑核苷酸序列的方法和组合物
CN116286823A (zh) * 2023-04-04 2023-06-23 尧唐(上海)生物科技有限公司 靶向TGFBI基因的sgRNA、载体及其应用
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US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US12359218B2 (en) 2017-07-28 2025-07-15 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US12435331B2 (en) 2017-03-10 2025-10-07 President And Fellows Of Harvard College Cytosine to guanine base editor
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
US12448461B2 (en) 2018-04-25 2025-10-21 Children's Medical Center Corporation ABCB5 ligands and substrates
US12473573B2 (en) 2013-09-06 2025-11-18 President And Fellows Of Harvard College Switchable Cas9 nucleases and uses thereof
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US20190185850A1 (en) * 2016-08-20 2019-06-20 Avellino Lab Usa, Inc. Single guide rna/crispr/cas9 systems, and methods of use thereof
JP7196104B2 (ja) * 2017-06-05 2022-12-26 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 増強された改変ウイルスカプシドタンパク質
JP7161730B2 (ja) * 2017-06-07 2022-10-27 国立大学法人 東京大学 顆粒状角膜変性症に対する遺伝子治療薬
US20190218532A1 (en) * 2017-09-19 2019-07-18 Massachusetts Institute Of Technology Streptococcus Canis Cas9 as a Genome Engineering Platform with Novel PAM Specificity
US11572574B2 (en) 2017-09-28 2023-02-07 Toolgen Incorporated Artificial genome manipulation for gene expression regulation
KR102670361B1 (ko) * 2017-09-28 2024-05-29 주식회사 툴젠 유전자 발현 조절을 위한 인위적인 게놈 조작
JP2021506251A (ja) 2017-12-14 2021-02-22 クリスパー セラピューティクス アーゲー 新規ｒｎａプログラム可能エンドヌクレアーゼ系、ならびにゲノム編集および他の適用におけるその使用
JP7144618B2 (ja) * 2018-12-20 2022-09-29 北京大学 バーコード付きガイドｒｎａ構築体を使用する効率的な遺伝子スクリーニングのための組成物及び方法
JP2023503618A (ja) * 2019-11-27 2023-01-31 ザ ジェネラル ホスピタル コーポレイション 遺伝子発現を活性化するためのシステムおよび方法
RU2730975C1 (ru) * 2020-02-18 2020-08-26 Ирина Викторовна Булыгина Способ лечения эндотелиально-эпителиальной дистрофии роговицы
WO2023004189A1 (en) * 2021-07-23 2023-01-26 Avellino Lab Usa, Inc. Method for detecting alleles associated with keratoconus

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