公开(公告)号：EP3416976A2

公开(公告)日：2018-12-26

申请号：EP17711024.4

申请日：2017-02-16

申请人： Yale University ,  Carnegie Mellon University

发明人： SALTZMAN, W., Mark ,  GLAZER, Peter ,  BAHAL, Raman ,  MCNEER, Nicole, Ali ,  LY, Danith, H. ,  QUIJANO, Elias

IPC分类号： C07K14/00

CPC分类号： C12N15/907 ,  A61K9/1647 ,  A61K48/005 ,  C07K14/003 ,  C12N15/102 ,  C12N15/11 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/15 ,  C12N2310/152 ,  C12N2310/318 ,  C12N2310/3181 ,  C12N2310/334 ,  C12N2310/351 ,  C12N2310/3519

摘要： Compositions and methods for enhancing targeted gene editing and methods of use thereof are disclosed. In the most preferred embodiments, gene editing is carried out utilizing a gene editing composition such as triplex-forming oligonucleotides, CRISPR, zinc finger nucleases, TALENS, or others, in combination with a gene modification potentiating agent such as stem cell factor (SCF), a CHK1 or ATR inhibitor, or a combination thereof. A particular preferred gene editing composition is triplex-forming peptide nucleic acids (PNAs) substituted at the γ position for increased DNA binding affinity. Nanoparticle compositions for intracellular delivery of the gene editing composition are also provided and particular advantageous for use with in vivo applications.

公开(公告)号：EP3356531A1

公开(公告)日：2018-08-08

申请号：EP16852730.7

申请日：2016-09-30

申请人： APSE LLC

发明人： ARHANCET, Juan, P. H.

IPC分类号： C12N15/113 ,  C12N15/82

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N15/8218 ,  C12N15/8286 ,  C12N2310/14 ,  C12N2310/15 ,  C12N2320/32 ,  C12N2330/50 ,  Y02A40/162

摘要： The invention describes recombinant DNA sequences transcribed into RNA constructs capable of forming pseudoknots and being encapsidated in Virus Like Particles having higher insect control efficacy than previously described RNA molecules.

公开(公告)号：EP1532246B1

公开(公告)日：2018-07-11

申请号：EP03753679.4

申请日：2003-09-01

申请人： Spark Therapeutics Ireland Limited

发明人： FARRAR, Gwenyth Jane ,  HUMPHRIES, Peter ,  KENNA, Paul Francis

IPC分类号： C12N15/113 ,  A61K48/00 ,  A01K67/027

CPC分类号： C12N15/113 ,  A01K67/0275 ,  A01K2207/15 ,  A01K2217/00 ,  A01K2217/05 ,  A01K2217/075 ,  A61K31/7105 ,  A61K31/711 ,  A61K38/00 ,  A61K45/06 ,  A61K48/00 ,  C12N2310/111 ,  C12N2310/12 ,  C12N2310/121 ,  C12N2310/14 ,  C12N2310/15 ,  C12N2310/53 ,  A61K2300/00

摘要： Methods and agents for suppressing expression of a mutant allele of a gene and providing a replacement nucleic acid are provided. The methods of the invention provide suppression effectors such as, for example, antisense nucleic acids, ribozymes, or RNAi, that bind to the gene or its RNA. The invention further provides for the introduction of a replacement nucleic acid with modified sequences such that the replacement nucleic acid is protected from suppression by the suppression effector. The replacement nucleic acid is modified at degenerate wobble positions in the target region of the suppression effector and thereby is not suppressed by the suppression effector. In addition, by altering wobble positions, the replacement nucleic acid can still encode a wild type gene product. The invention has the advantage that the same suppression strategy could be used to suppress, in principle, many mutations in a gene. Also disclosed is a transgenic mouse that expresses human rhodopsin (modified replacement gene) and a transgenic mouse that expresses a suppression effector targeting rhodopsin. Also disclosed in intraocular administration of siRNA.

公开(公告)号：EP2258848B1

公开(公告)日：2014-03-05

申请号：EP10009491.1

申请日：2000-12-20

申请人： Genentech, Inc.

发明人： Chen, Jian ,  Filvaroff, Ellen ,  Fong, Sherman ,  Goddard, Audrey ,  Godowski, Paul J. ,  Grimaldi, Christopher J. ,  Gurney, Austin L. ,  Li, Hanzhong ,  Hillan, Kenneth J. ,  Tumas, Daniel ,  Van Lookeren, Menno ,  Vandlen, Richard L. ,  Watanabe, Colin K. ,  Williams, P., Mickey ,  Wood, William I. ,  Yansura, Daniel G.

IPC分类号： C12N15/12 ,  C07K14/715 ,  C07K14/54 ,  C12N15/62 ,  C07K16/24 ,  C07K16/28 ,  A61K31/70 ,  A61K38/17 ,  A61K38/20 ,  A61K39/395 ,  G01N33/566

CPC分类号： A61K38/20 ,  C07K14/54 ,  C07K14/7155 ,  C12N15/1136 ,  C12N2310/11 ,  C12N2310/12 ,  C12N2310/15

公开(公告)号：EP2419535A1

公开(公告)日：2012-02-22

申请号：EP10764798.4

申请日：2010-03-15

申请人： Northwestern University

发明人： MIRKIN, Chad, A. ,  PALLER, Amy, S. ,  GILJOHANN, David, A.

IPC分类号： C12Q1/68

CPC分类号： A61K31/7088 ,  A61K9/0014 ,  A61K9/0019 ,  A61K47/6923 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/15 ,  C12N2320/32

摘要： The present invention relates to compositions and methods for delivering an oligonucleotide-functionalized nanoparticle.

公开(公告)号：EP1897945B1

公开(公告)日：2012-01-18

申请号：EP07016901.6

申请日：2000-12-20

申请人： Genentech, Inc.

发明人： Chen, Jian ,  Filvaroff, Ellen ,  Fong, Sherman ,  Goddard, Audrey ,  Godowski, Paul J. ,  Grimaldi, Christopher J. ,  Gurney, Austin L. ,  Li, Hanzhong ,  Hillan, Kenneth J. ,  Tumas, Daniel ,  Van Lookeren, Menno ,  Vandlen, Richard L. ,  Watanabe, Colin K. ,  Williams, P. Mickey ,  Wood, William I. ,  Yansura, Daniel G.

IPC分类号： C12N15/12 ,  C07K14/715 ,  C07K14/54 ,  C12N15/62 ,  C07K16/24 ,  C07K16/28 ,  A61K31/70 ,  A61K38/17 ,  A61K38/20 ,  A61K39/395 ,  G01N33/566

CPC分类号： A61K38/20 ,  C07K14/54 ,  C07K14/7155 ,  C12N15/1136 ,  C12N2310/11 ,  C12N2310/12 ,  C12N2310/15

公开(公告)号：EP2290081A2

公开(公告)日：2011-03-02

申请号：EP10009490.3

申请日：2000-12-20

申请人： Genentech, Inc.

发明人： Chen, Jian ,  Filvaroff, Ellen ,  Fong, Sherman ,  Goddard, Audrey ,  Godowski, Paul J. ,  Grimaldi, Christopher J. ,  Gurney, Austin L. ,  Li, Hanzhong ,  Hillan, Kenneth J. ,  Tumas, Daniel ,  Van Lookeren, Menno ,  Vandlen, Richard L. ,  Watanabe, Colin K. ,  Williams, P., Mickey ,  Wood, William I. ,  Yansura, Daniel G.

IPC分类号： C12N15/12 ,  C07K14/715 ,  C07K14/54 ,  C12N15/62 ,  C07K16/24 ,  C07K16/28 ,  A61K31/70 ,  A61K38/17 ,  A61K38/20 ,  A61K39/395 ,  G01N33/566

CPC分类号： A61K38/20 ,  C07K14/54 ,  C07K14/7155 ,  C12N15/1136 ,  C12N2310/11 ,  C12N2310/12 ,  C12N2310/15

摘要： The present invention is directed to novel polypeptides and to nucleic acid molecules encoding those polypeptides. Also provided herein a re vectors and host cells comprising those nucleic acid sequences, chimeric polypeptide molecules comprising the polypeptides of the present invention fused to heterologous polypeptide sequences, antibodies which bind to the polypeptides of the present invention and to methods for producing the polypeptides of the present invention.

摘要翻译： 本发明涉及新型多肽和编码那些多肽的核酸分子。 本文还提供了包含这些核酸序列的重组载体和宿主细胞，包含与异源多肽序列融合的本发明多肽的嵌合多肽分子，与本发明多肽结合的抗体以及产生本发明多肽的方法 本发明。

公开(公告)号：EP2015782A4

公开(公告)日：2010-04-07

申请号：EP07760600

申请日：2007-04-12

申请人： CROSSLINK GENETICS CORP

发明人： STANOJEVIC DUSAN

IPC分类号： C12Q1/68 ,  A61K31/00 ,  A61K38/00 ,  C12Q1/02

CPC分类号： C12N15/111 ,  C12N15/1135 ,  C12N2310/11 ,  C12N2310/15 ,  C12N2310/3513

公开(公告)号：EP0954585B1

公开(公告)日：2009-11-25

申请号：EP97910785.1

申请日：1997-10-01

申请人： GERON CORPORATION

发明人： MORIN, Gregg, B. ,  ANDREWS, William, H.

IPC分类号： C12N15/54 ,  C12N9/12 ,  C12Q1/68 ,  C12Q1/48 ,  C12N15/11 ,  C12N15/85 ,  A01K67/027 ,  C07K16/40 ,  A61K38/45 ,  A61K31/70 ,  C12N1/21 ,  C12N1/19

CPC分类号： C12N15/113 ,  A01K2217/05 ,  A61K38/00 ,  A61K39/00 ,  A61K48/00 ,  C07K2319/00 ,  C12N9/1241 ,  C12N9/1276 ,  C12N15/1137 ,  C12N2310/15 ,  C12N2310/315 ,  C12Y207/07049 ,  Y10S530/827 ,  Y10S530/828

摘要： The invention provides compositions and methods related to human telomerase reverse transcriptase (hTRT), the catalytic protein subunit of human telomerase. The polynucleotides and polypeptides of the invention are useful for diagnosis, prognosis and treatment of human diseases, for changing the proliferative capacity of cells and organisms, and for identification and screening of compounds and treatments useful for treatment of diseases such as cancers.

公开(公告)号：EP1941036A1

公开(公告)日：2008-07-09

申请号：EP06793134.5

申请日：2006-09-01

申请人： LVMH RECHERCHE ,  Centre National de la Recherche Scientifique (CNRS) Etablissement Public à Caractère Scientifique et Technologique ,  MUSEUM NATIONAL D'HISTOIRE NATURELLE

发明人： KURFÜRST, Robin ,  BONNET-DUQUENNOY, Mathilde ,  LAZOU, Kristell ,  GIOVANNANGELI, Carine

IPC分类号： C12N15/11 ,  A61Q17/04 ,  A61Q19/02 ,  A61P17/00 ,  A61K31/7088

CPC分类号： C12N15/1137 ,  A61K8/606 ,  A61Q17/04 ,  A61Q19/02 ,  C12N2310/15 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12Y114/18001

摘要： The invention concerns an antigenic oilgonucleotide hybridizing specifically with the gene encoding human tyrosinase through Hoogsteen pairing between complementary bases, said oligonucleotide forming a triple-helix structure with the human tyrosinase gene. The invention also concerns the use of said oligonucleotide as depigmenting or skin-whitening agent in a cosmetic composition or in a dermatological composition.