[특허]Methods and sequences to preferentially suppress expression of mutated huntingtin

[미국특허] Methods and sequences to preferentially suppress expression of mutated huntingtin 원문보기

IPC분류정보
국가/구분	United States(US) Patent 등록
국제특허분류(IPC7판)	C12Q-001/68 C07H-021/04 C12P-019/34
출원번호	US-0560178 (2009-09-15)
등록번호	US-9133517 (2015-09-15)
발명자 / 주소	Van Bilsen, Paul H. J. Jaspers, Leonie
출원인 / 주소	Medtronics, Inc.
대리인 / 주소	Fox Rothschild LLP
인용정보	피인용 횟수 : 0 인용 특허 : 57

초록 ▼

Disclosed herein are methods and sequences to preferentially suppress the expression of the mutated huntingtin (“htt”) protein over expression of the normal htt protein. Also disclosed are methods comprising screening an individual for the heterozygous presence of one or more single nucleotide polymorphisms within the individual's Huntington's genes; administering nucleic acid molecules comprising nucleotide sequences that preferentially suppress the expression of amino acid sequences encoding for mutated huntingtin (“htt”) over suppressing the expression of amino acid sequences encoding for normal htt by targeting an area of a Huntington's disease gene that is heterozygous for the presence of one or more single nucleotide polymorphisms.

대표청구항 ▼

1. A kit for screening an individual for the heterozygous presence of one or more single nucleotide polymorphisms (SNPs) within said individual's Huntington's disease genes, the kit comprising two pairs of molecular beacons selected from the group consisting of: (a) a molecular beacon that detects a single polymorphism found in SEQ ID NO: 13, wherein said molecular beacon comprises at least a portion of a reverse complement of SEQ ID NO: 13 and a molecular beacon that detects a single polymorphism found in SEQ ID NO: 14, wherein said molecular beacon comprises at least a portion of a reverse complement of SEQ ID NO: 14;(b) a molecular beacon that detects a single polymorphism found in SEQ ID NO: 15, wherein said molecular beacon comprises at least a portion of a reverse complement of SEQ ID NO: 15 and a molecular beacon that detects a single polymorphism found in SEQ ID NO: 16, wherein said molecular beacon comprises at least a portion of a reverse complement of SEQ ID NO: 16;(c) a molecular beacon that detects a single polymorphism found in SEQ ID NO: 3, wherein said molecular beacon comprises at least a portion of a reverse complement of SEQ ID NO: 3; and a molecular beacon that detects a single polymorphism found in SEQ ID NO: 4, wherein said molecular beacon comprises at least a portion of a reverse complement of SEQ ID NO: 4; and(d) a molecular beacon that detects a single polymorphism found in SEQ ID NO: 7, wherein said molecular beacon comprises at least a portion of a reverse complement of SEQ ID NO: 7 and a molecular beacon that detects a single polymorphism found in SEQ ID NO: 8, wherein said molecular beacon comprises at least a portion of a reverse complement of SEQ ID NO: 8, wherein all the molecular beacons that detect a SNP for which said individual is heterozygous are selected from the group. 2. The kit of claim 1, further comprising nucleic acid molecules capable of mediating RNA interference (RNAi) that preferentially suppress the expression of amino acid sequences encoding for mutated huntingtin (“htt”) over suppressing the expression of amino acid sequences encoding for normal htt by targeting an area of a Huntington's disease gene that is heterozygous for the presence of one or more single nucleotide polymorphisms. 3. The kit of claim 1 wherein said one or more single nucleotide polymorphisms are selected from the group consisting of SNP8, SNP7 SNP2, and SNP4. 4. A method for screening individuals for the heterozygous presence of one or more SNPs within the individual's Huntingtin genes, comprising probing a nucleic sequence encoding huntingtin of said individual with one or more molecular beacons of kit of claim 1, wherein said one or more SNPs are selected from SNP2, SNP4, SNP7 and SNP8. 5. A kit for screening an individual for the heterozygous presence of one or more single nucleotide polymorphisms (SNPs) within said individual's Huntington's disease genes, the kit comprising: a) a molecular beacon that detects a single polymorphism found in SEQ ID NO: 13, wherein said molecular beacon comprises at least a portion of a reverse complement of SEQ ID NO: 13;b) a molecular beacon that detects a single polymorphism found in SEQ ID NO: 14, wherein said molecular beacon comprises at least a portion of a reverse complement of SEQ ID NO: 14;c) a molecular beacon that detects a single polymorphism found in SEQ ID NO: 15, wherein said molecular beacon comprises at least a portion of a reverse complement of SEQ ID NO: 15;d) a molecular beacon that detects a single polymorphism found in SEQ ID NO: 16, wherein said molecular beacon comprises at least a portion of a reverse complement of SEQ ID NO: 16; ande) nucleic acid molecules capable of mediating RNAi that preferentially suppress the expression of amino acid sequences encoding for mutated huntingtin (“htt”) over suppressing the expression of amino acid sequences encoding for normal htt by targeting an area of a Huntington's disease gene that is heterozygous for the presence of one or more single nucleotide polymorphisms selected from the group consisting of SNP07 and SNP08, wherein all the molecular beacons that detect a SNP for which said individual is heterozygous are selected from the group consisting of a)-d). 6. A method for screening individuals for the heterozygous presence of one or more SNPs within the individual's Huntingtin genes, comprising probing a nucleic sequence encoding huntingtin of said individual with one or more molecular beacons of kit of claim 5, wherein said one or more SNPs are selected from SNP7 and SNP8. 7. The kit of claim 1, wherein the kit comprises said pairs (a) and (b) of molecular beacons. 8. The kit of claim 7, wherein the kit further comprises at least one pair of molecular beacons selected from said pairs (c) and (d). 9. The kit of claim 1, wherein said each SNP detected by each molecular beacon in the kit accounts for 9% or more heterozygous presence in the Huntington's disease gene in a Huntington's disease patient population. 10. The kit of claim 5, wherein said each SNP detected by each molecular beacon in the kit accounts for 9% or more heterozygous presence in the Huntington's disease gene in a Huntington's disease patient population.

이 특허에 인용된 특허 (57) 인용/피인용 타임라인 분석

Kaplitt Michael G. ; During Matthew J., AAV-mediated delivery of DNA to cells of the nervous system.
상세보기
John A. Chiorini ; Robert M. Kotin ; Beverly Davidson, AAV4 vector and uses thereof.
상세보기
Powell, David J.; Chapman, Conrad G.; Murphy, Kay; Smith, Trudi S., ASP2.
상세보기
John F. Engelhardt ; Dongsheng Duan, Adeno-associated virus vectors.
상세보기
Kumar Vijaya B., Antisense modulation of amyloid beta protein expression.
상세보기
Taheri, Syde A., Autologous myocyte micro granual retrieval and implantation (AMMGRI).
상세보기
Elsberry, Dennis D.; Christiansen, Chris; Morris, Mary M.; Hankner, Douglas Owen; Hamlen, Robert Cushing, Catheter for target specific drug delivery.
상세보기
Morris, Mary M.; Hankner, Douglas Owen, Catheter for target specific drug delivery.
상세보기
Sah,Dinah Wen Yee; Hadwiger,Philipp; Roehl,Ingo; Bramlage,Birgit; Tan,Pamela; Vornlocher,Hans Peter; Bumcrot,David, Compositions and methods for inhibiting expression of Huntingtin gene.
상세보기
Burns James W. ; Greenawalt Keith E. ; Masi Louis, Compositions containing polyanionic polysaccharides and hydrophobic bioabsorbable polymers.
상세보기
Laske Douglas W. ; Oldfield Edward H. ; Bobo Richard Hunt ; Dedrick Robert L. ; Morrison Paul F., Convection-enhanced drug delivery.
상세보기
Knight Richard ; Barsoum Michel W., Corrosion, oxidation and/or wear-resistant coatings.
상세보기
Baetge Edward E. (Barrington RI) Hammang Joseph P. (Barrington RI) Gentile Frank T. (Warwick RI) Lindner Mark D. (Bristol RI) Winn Shelley R. (Smithfield RI) Emerich Dwaine F. (Providence RI), Delivery of biologically active molecules using cells contained in biocompatible immunoisolatory capsules.
상세보기
Paola Leone ; Matthew J. During ; Frank L. Sorgi, Delivery system for gene therapy to the brain.
상세보기
Hayakawa Toru,JPX ; Yoshimine Toshiki,JPX ; Yamamoto Hiromu,JPX ; Sato Akira,JPX ; Irie Tsunemasa,JPX ; Fujioka Keiji,JPX ; Takada Yoshihiro,JPX ; Sasaki Yoshio,JPX, Equipment for intracerebral administration of preparations.
상세보기
Kimberly Robert P. ; Edberg Jeffrey C., FC receptor polymorphism.
상세보기
John H. Lawrence, III ; J. Kevin Donahue, Gene delivery compositions and methods.
상세보기
Kleinerman George J. (King\s Lynn GB2) Woodcock John M. (King\s Lynn GB2), Gloves.
상세보기
Noonberg Sarah B. (Berkeley CA) Hunt C. Anthony (San Francisco CA), In vivo oligonucleotide generator, and methods of testing the binding affinity of triplex forming oligonucleotides deriv.
상세보기
Elsberry Dennis D., Intraparenchymal infusion catheter system.
상세보기
Elsberry, Dennis D., Intraparenchymal infusion catheter system.
상세보기
Robert L. Werner ; Thomas J. Winkler ; Ray Gravlin, Medical lead adaptor.
상세보기
Castle Vernon P. ; Nelson Jerrold ; Gullickson George W. ; Skubitz Frank L., Medical lead adaptor for external medical device.
상세보기
Soykan Orhan ; Donovan Maura G., Method and system for myocardial identifier repair.
상세보기
Tsuji Shoji,JPX ; Sanpei Kazuhiro,JPX, Method for diagnosing spinocerebellar ataxia type 2 and primers therefor.
상세보기
Toomas Neuman ; Kikuo Suda JP; Howard O. Nornes, Method for inducing DNA synthesis in neurons.
상세보기
Lo, Yuk-Ming Dennis; Chiu, Rossa Wai Kwun; Tsui, Bo Yin; Ding, Chunming; Cantor, Charles, Method for the detection of chromosomal aneuploidies.
상세보기
Mandel Ronald J.,SEX ; Leff Stuart E., Method of controlling L-Dopa production and of treating dopamine deficiency.
상세보기
Nakayama Takahiro,JPX ; Kageyama Nobuo,JPX ; Chikaso Takashi,JPX, Method of making a silicon carbide product.
상세보기
Bankiewicz Krys ; Cunningham Janet, Methods of treating Parkinson's disease using recombinant adeno-associated vector (rAAV).
상세보기
Gruber Harry E. (San Diego CA) Miller Leonard P. (Carlsbad CA), Methods of treating injury to the central nervous system.
상세보기
Gluckman Peter D.,NZX ; Williams Christopher E.,NZX ; Guan Jian,NZX, Methods to improve neural outcome.
상세보기
Morris Christopher Miles,GBX, Model for chronic cerebral inflammation by intracerebral injection of double stranded RNA.
상세보기
Unger, Gretchen M., Nanoparticle encapsulation system and method.
상세보기
Gill, Steven, Neurosurgical guide device.
상세보기
William M. Pardridge, Non-invasive gene targeting to the brain.
상세보기
Fogarty Patrick ; Lipsick Joseph, P element derived vector and methods for its use.
상세보기
Dunn Richard L. (Fort Collins CO) Tipton Arthur J. (Fort Collins CO), Polymeric compositions useful as controlled release implants.
상세보기
Liou Derlin (4 Fl. ; No. 293 ; Sung-Chiang Rd. Taipei TWX), Powerfree glove and its making method.
상세보기
Mullis Kary B. (Kensington CA), Process for amplifying nucleic acid sequences.
상세보기
Mullis Kary B. (La Jolla CA) Erlich Henry A. (Oakland CA) Arnheim Norman (Woodland Hills CA) Horn Glenn T. (Emeryville CA) Saiki Randall K. (Richmond CA) Scharf Stephen J. (Berkeley CA), Process for amplifying, detecting, and/or cloning nucleic acid sequences.
상세보기
Mullis Kary B. (La Jolla CA) Erlich Henry A. (Oakland CA) Gelfand David H. (Oakland CA) Horn Glenn (Emeryville CA) Saiki Randall K. (Richmond CA), Process for amplifying, detecting, and/or cloning nucleic acid sequences using a thermostable enzyme.
상세보기
Mullis Kary B. (Kensington CA) Erlich Henry A. (Oakland CA) Arnheim Norman (Woodland Hills CA) Horn Glenn T. (Emeryville CA) Saiki Randall K. (Richmond CA) Scharf Stephen J. (Berkeley CA), Process for amplifying, detecting, and/or-cloning nucleic acid sequences.
상세보기
Tamer El-Raghy ; Michel W. Barsoum ; Mats Sundberg SE; Hans Pettersson SE, Process for forming 312 phase materials and process for sintering the same.
상세보기
Barsoum Michel W. ; El-Raghy Tamer, Process for making a dense ceramic workpiece.
상세보기
Comben Richard H. (St. Paul MN) Grant Barry J. (Roberts WI) Serfling Michael D. (St. Louis Park MN) Cross ; Jr. Thomas E. (St. Francis MN) Stanton David J. (Anoka MN), Screening cable connector for interface to implanted lead.
상세보기
Hook Vivian Y. H., Secretases related to Alzheimer's dementia.
상세보기
Hook Vivian Y. H., Secretases related to alzheimer's dementia.
상세보기
Barsoum Michael ; El-Raghy Tamer ; Brodkin Dmitri ; Zavaliangos Antonios ; Kalidindi Surya, Synthesis of 312 phases and composites thereof.
상세보기
Elsberry Dennis D. ; Rise Mark T., Techniques for treating neurodegenerative disorders by infusion of nerve growth factors into the brain.
상세보기
Elsberry Dennis D. ; Rise Mark T., Techniques of treating neurodegenerative disorders by brain infusion.
상세보기
Elsberry Dennis D. ; Rise Mark T., Techniques of treating neurodegenerative disorders by brain infusion.
상세보기
March Keith L. ; Aita Michael ; Kesten Randy ; Smith Craig, Therapeutic and diagnostic agent delivery.
상세보기
March Keith L. ; Aita Michael ; Kesten Randy ; Smith Craig, Therapeutic and diagnostic agent delivery.
상세보기
Ellis Louis ; Hendrickson Gary L., Transmyocardial revascularization catheter and method.
상세보기
Mickle Donald A. G.,CAXITX M5M 2X9 ; Li Ren-Ke,CAXITX M1V 1B4 ; Weisel Richard D.,CAXITX M4X 1S8, Transplants for myocardial scars and methods and cellular preparations.
상세보기
Boyce Frederick M., Use of a non-mammalian DNA virus to express an exogenous gene in a mammalian cell.
상세보기

활용도 분석정보

상세보기

다운로드

내보내기

활용도 Top5 특허

해당 특허가 속한 카테고리에서 활용도가 높은 상위 5개 콘텐츠를 보여줍니다.
더보기 버튼을 클릭하시면 더 많은 관련자료를 살펴볼 수 있습니다.

IPC	Description
A	생활필수품
A62	인명구조; 소방(사다리 E06C)
A62B	인명구조용의 기구, 장치 또는 방법(특히 의료용에 사용되는 밸브 A61M 39/00; 특히 물에서 쓰이는 인명구조 장치 또는 방법 B63C 9/00; 잠수장비 B63C 11/00; 특히 항공기에 쓰는 것, 예. 낙하산, 투출좌석 B64D; 특히 광산에서 쓰이는 구조장치 E21F 11/00)
A62B-1/08	.. 윈치 또는 풀리에 제동기구가 있는 것

내보내기 구분	파일저장 인쇄 메일전송
구성항목	기본정보 상세정보 관리번호, 국가코드, 자료구분, 상태, 출원번호, 출원일자, 공개번호, 공개일자, 등록번호, 등록일자, 발명명칭(한글), 발명명칭(영문), 출원인(한글), 출원인(영문), 출원인코드, 대표IPC 관리번호, 국가코드, 자료구분, 상태, 출원번호, 출원일자, 공개번호, 공개일자, 공고번호, 공고일자, 등록번호, 등록일자, 발명명칭(한글), 발명명칭(영문), 출원인(한글), 출원인(영문), 출원인코드, 대표출원인, 출원인국적, 출원인주소, 발명자, 발명자E, 발명자코드, 발명자주소, 발명자 우편번호, 발명자국적, 대표IPC, IPC코드, 요약, 미국특허분류, 대리인주소, 대리인코드, 대리인(한글), 대리인(영문), 국제공개일자, 국제공개번호, 국제출원일자, 국제출원번호, 우선권, 우선권주장일, 우선권국가, 우선권출원번호, 원출원일자, 원출원번호, 지정국, Citing Patents, Cited Patents
저장형식	Text(ASCII format) Excel format PIAS분석(.xls)
메일정보	받는사람 (필수) @ 보내는사람 (선택) @ 제목 내용 KISTI 검색결과 이메일 서비스
안내	총 건의 자료가 검색되었습니다. 다운받으실 자료의 인덱스를 입력하세요. (1-10,000) 검색결과의 순서대로 최대 10,000건 까지 다운로드가 가능합니다. 데이타가 많을 경우 속도가 느려질 수 있습니다.(최대 2~3분 소요) 다운로드 파일은 UTF-8 형태로 저장됩니다. 파일의 내용이 제대로 보이지 않을실 때는 웹브라우저 상단의 보기 -> 인코딩 -> 자동선택 여부를 확인하십시오. ~ Text(ASCII format) Excel format

연합인증

[미국특허] Methods and sequences to preferentially suppress expression of mutated huntingtin 원문보기

초록 ▼

대표청구항 ▼

연구과제 타임라인

이 특허에 인용된 특허 (57) 인용/피인용 타임라인 분석

활용도 분석정보

활용도 Top5 특허

관련 콘텐츠

특허 원문 보기

IPC 상위 출원인

AI-Helper ※ AI-Helper는 오픈소스 모델을 사용합니다.

선택된 텍스트

연합인증

[미국특허] Methods and sequences to preferentially suppress expression of mutated huntingtin 원문보기

초록 ▼

대표청구항 ▼

연구과제 타임라인

전체(0) 논문(0) 특허(0) 보고서(0)

전체(0) 논문(0) 특허(0) 보고서(0)

이 특허에 인용된 특허 (57) 인용/피인용 타임라인 분석

활용도 분석정보

활용도 Top5 특허 더보기

관련 콘텐츠

특허 원문 보기

IPC 상위 출원인

AI-Helper ※ AI-Helper는 오픈소스 모델을 사용합니다.

선택된 텍스트

활용도 Top5 특허