US6924128B2
(en)
|
1994-12-06 |
2005-08-02 |
Targeted Genetics Corporation |
Packaging cell lines for generation of high titers of recombinant AAV vectors
|
DE69841982D1
(de)
*
|
1997-03-14 |
2010-12-16 |
Philadelphia Children Hospital |
Zusammensetzungen zum einsatz in einer gentherapie zur behandlung von hämophilie
|
US6566118B1
(en)
|
1997-09-05 |
2003-05-20 |
Targeted Genetics Corporation |
Methods for generating high titer helper-free preparations of released recombinant AAV vectors
|
US6989264B2
(en)
|
1997-09-05 |
2006-01-24 |
Targeted Genetics Corporation |
Methods for generating high titer helper-free preparations of released recombinant AAV vectors
|
US6953690B1
(en)
|
1998-03-20 |
2005-10-11 |
The Trustees Of The University Of Pennsylvania |
Compositions and methods for helper-free production of recombinant adeno-associated viruses
|
JP2002506652A
(ja)
*
|
1998-03-20 |
2002-03-05 |
トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア |
組換えアデノ随伴ウイルスのヘルパーを含まない製造のための組成物及び方法
|
US6436392B1
(en)
*
|
1998-05-20 |
2002-08-20 |
University Of Iowa Research Foundation |
Adeno-associated virus vectors
|
EP1849872A1
(en)
*
|
1998-05-20 |
2007-10-31 |
University Of Iowa Research Foundation |
Adeno-associated virus vectors and uses thereof
|
DE19905501B4
(de)
|
1999-02-10 |
2005-05-19 |
MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie |
Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels
|
JP4693244B2
(ja)
|
1999-03-18 |
2011-06-01 |
ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア |
組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
|
US6258595B1
(en)
*
|
1999-03-18 |
2001-07-10 |
The Trustees Of The University Of Pennsylvania |
Compositions and methods for helper-free production of recombinant adeno-associated viruses
|
US6893865B1
(en)
|
1999-04-28 |
2005-05-17 |
Targeted Genetics Corporation |
Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
|
WO2000075365A2
(en)
|
1999-06-08 |
2000-12-14 |
University Of Iowa Research Foundation |
Compounds and methods to enhance raav transduction
|
US7122335B1
(en)
|
1999-06-08 |
2006-10-17 |
University Of Iowa Research Foundation |
Compounds and methods to enhance rAAV transduction
|
JP2003533170A
(ja)
*
|
1999-10-07 |
2003-11-11 |
ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン |
アデノ随伴ウイルスおよびその使用
|
US7241447B1
(en)
|
1999-10-07 |
2007-07-10 |
University Of Iowa Research Foundation |
Adeno-associated virus vectors and uses thereof
|
DE60139394D1
(de)
*
|
2000-04-28 |
2009-09-10 |
Asklepios Biopharmaceutical In |
Für das dystrophin-minigen kodierende dna-sequenzen und verfahren zu deren verwendung
|
CA2406743A1
(en)
|
2000-04-28 |
2001-11-08 |
The Trustees Of The University Of Pennsylvania |
Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
|
US8241622B2
(en)
|
2001-07-13 |
2012-08-14 |
University Of Iowa Research Foundation |
Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
|
PL222683B1
(pl)
|
2001-11-13 |
2016-08-31 |
Univ Pennsylvania |
Rekombinowane wirusy stowarzyszone z adenowirusem (AAV), sposoby ich wytwarzania, pakująca komórka gospodarza, kompozycja zawierająca wirus, zastosowanie wirusa, wyizolowane wirusy AAV, białka,sztuczne białka kapsydu, cząsteczki, komórki gospodarza, sposoby dostarczania transgenu, sposób identyfikacji serotypu, zestaw diagnostyczny, sposób izolacji nowych wirusów, rekombinowana komórka
|
EP1463805B1
(en)
|
2001-12-17 |
2014-10-22 |
The Trustees of The University of Pennsylvania |
Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
|
PT1453547T
(pt)
|
2001-12-17 |
2016-12-28 |
Univ Pennsylvania |
Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
|
AU2004227915A1
(en)
|
2003-03-31 |
2004-10-21 |
John F. Engelhardt |
Pharmico-gene theraphy of epithelial sodium channel associated disoders
|
CN102199626B
(zh)
|
2003-09-30 |
2015-06-24 |
宾夕法尼亚大学托管会 |
腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
|
EP1742657B1
(en)
|
2004-04-28 |
2013-11-06 |
The Trustees of The University of Pennsylvania |
Immunization regimen with e4-deleted adenovirus prime and e1-deleted adenovirus boost
|
US8394386B2
(en)
|
2004-04-28 |
2013-03-12 |
The Trustees Of The University Of Pennsylvania |
Sequential delivery of immunogenic molecules via adenovirus and adeno-associated virus-mediated administrations
|
CN1286981C
(zh)
*
|
2004-11-30 |
2006-11-29 |
华中科技大学同济医学院附属同济医院 |
表达人类cyp2j2反义基因的重组腺相关病毒及其制备方法
|
CN100374572C
(zh)
*
|
2005-07-19 |
2008-03-12 |
北京市肿瘤防治研究所 |
一种体外培养骨骼肌表达体系
|
ES2569365T3
(es)
|
2006-11-29 |
2016-05-10 |
Nationwide Children's Hospital |
Inhibición de miostatina para la potenciación de músculo y/o la mejora de la función muscular
|
US11219696B2
(en)
|
2008-12-19 |
2022-01-11 |
Nationwide Children's Hospital |
Delivery of polynucleotides using recombinant AAV9
|
US9415121B2
(en)
|
2008-12-19 |
2016-08-16 |
Nationwide Children's Hospital |
Delivery of MECP2 polynucleotide using recombinant AAV9
|
CN108744262A
(zh)
|
2010-11-23 |
2018-11-06 |
普莱萨格生命科学公司 |
用于实体递送的治疗方法和组合物
|
US10196636B2
(en)
|
2011-04-21 |
2019-02-05 |
Nationwide Children's Hospital, Inc. |
Recombinant virus products and methods for inhibition of expression of myotilin
|
ES2702496T3
(es)
|
2011-04-21 |
2019-03-01 |
Nationwide Childrens Hospital Inc |
Productos de virus recombinante y procedimientos para la inhibición de la expresión de la miotilina
|
US20130039888A1
(en)
|
2011-06-08 |
2013-02-14 |
Nationwide Children's Hospital Inc. |
Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
|
WO2013016352A1
(en)
|
2011-07-25 |
2013-01-31 |
Nationwide Children's Hospital, Inc. |
Recombinant virus products and methods for inhibition of expression of dux4
|
US9434928B2
(en)
|
2011-11-23 |
2016-09-06 |
Nationwide Children's Hospital, Inc. |
Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
|
US20150252384A1
(en)
|
2012-08-01 |
2015-09-10 |
National Children's Hospital |
Intrathecal delivery of recombinant adeno-associated virus 9
|
EP2895606A4
(en)
|
2012-09-17 |
2016-07-06 |
Res Inst Nationwide Childrens Hospital |
COMPOSITIONS AND METHOD FOR THE TREATMENT OF AMYOTROPHER LATERAL SCLEROSIS (ALS)
|
ES2959747T3
(es)
|
2013-02-15 |
2024-02-28 |
Bioverativ Therapeutics Inc |
Gen del factor VIII optimizado
|
JP6461917B2
(ja)
|
2013-04-20 |
2019-01-30 |
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 |
エクソン2標的U7snRNAポリヌクレオチド構築物の組換えアデノ随伴ウイルスによる送達
|
RS63942B1
(sr)
|
2013-08-27 |
2023-02-28 |
Res Inst Nationwide Childrens Hospital |
Proizvodi i postupci za lečenje amiotrofične lateralne skleroze
|
EP3065784A4
(en)
|
2013-11-05 |
2017-05-10 |
The Research Institute at Nationwide Children's Hospital |
COMPOSITIONS AND METHODS FOR INHIBITING NF- kB AND SOD-1 TO TREAT AMYOTROPHIC LATERAL SCLEROSIS
|
PL3116900T3
(pl)
|
2014-03-09 |
2021-03-08 |
The Trustees Of The University Of Pennsylvania |
Kompozycje użyteczne w leczeniu niedoboru transkarbamylazy ornitynowej (otc)
|
WO2015191508A1
(en)
|
2014-06-09 |
2015-12-17 |
Voyager Therapeutics, Inc. |
Chimeric capsids
|
WO2016004113A1
(en)
|
2014-06-30 |
2016-01-07 |
Biogen Ma Inc. |
Optimized factor ix gene
|
EP3572516A1
(en)
|
2014-08-09 |
2019-11-27 |
The Research Institute at Nationwide Children's Hospital |
Methods and materials for activating an internal ribosome entry site in exon 5 of the dmd gene
|
WO2016057975A2
(en)
|
2014-10-10 |
2016-04-14 |
Research Institute At Nationwide Children's Hospital |
Guided injections for aav gene transfer to muscle
|
BR112017009497A2
(pt)
|
2014-11-05 |
2018-02-06 |
Voyager Therapeutics, Inc. |
polinucleotídeos de aadc para o tratamento da doença de parkinson
|
EP3215602B1
(en)
|
2014-11-05 |
2019-12-25 |
The Research Institute at Nationwide Children's Hospital |
Methods and materials for producing recombinant viruses in eukaryotic microalgae
|
US10597660B2
(en)
|
2014-11-14 |
2020-03-24 |
Voyager Therapeutics, Inc. |
Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
|
ES2878451T3
(es)
|
2014-11-14 |
2021-11-18 |
Voyager Therapeutics Inc |
Polinucleótidos moduladores
|
EP3230441A4
(en)
|
2014-12-12 |
2018-10-03 |
Voyager Therapeutics, Inc. |
Compositions and methods for the production of scaav
|
CA2998636A1
(en)
|
2015-09-17 |
2017-03-23 |
Research Institute At Nationwide Children's Hospital |
Methods and materials for galgt2 gene therapy
|
US10548947B2
(en)
|
2015-11-05 |
2020-02-04 |
Bamboo Therapeutics, Inc. |
Modified Friedreich ataxia genes and vectors for gene therapy
|
CA3006569A1
(en)
|
2015-12-02 |
2017-06-08 |
Voyager Therapeutics, Inc. |
Assays for the detection of aav neutralizing antibodies
|
HRP20221089T1
(hr)
|
2016-02-01 |
2022-11-25 |
Bioverativ Therapeutics Inc. |
Optimizirani geni faktora viii
|
WO2017139381A1
(en)
|
2016-02-08 |
2017-08-17 |
University Of Iowa Research Foundation |
Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
|
JP6966463B2
(ja)
|
2016-02-26 |
2021-11-17 |
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 |
組換えウイルス産物及びdux4エクソンスキッピングを誘導するための方法
|
US11684679B2
(en)
|
2016-03-07 |
2023-06-27 |
University Of Iowa Research Foundation |
AAV-mediated expression using a synthetic promoter and enhancer
|
WO2017173411A1
(en)
|
2016-04-02 |
2017-10-05 |
Research Institute At Nation Wide Children's Hospital |
Modified u6 promoter system for tissue specific expression
|
MA45477A
(fr)
|
2016-04-15 |
2019-02-20 |
Res Inst Nationwide Childrens Hospital |
Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
|
MY195439A
(en)
|
2016-04-15 |
2023-01-21 |
Res Inst Nationwide Childrens Hospital |
Adeno-Associated Virus Vector Delivery of B-Sarcoglycan and Microrna-29 and the Treatment of Muscular Dystrophy
|
US11326182B2
(en)
|
2016-04-29 |
2022-05-10 |
Voyager Therapeutics, Inc. |
Compositions for the treatment of disease
|
WO2017189964A2
(en)
|
2016-04-29 |
2017-11-02 |
Voyager Therapeutics, Inc. |
Compositions for the treatment of disease
|
US11951121B2
(en)
|
2016-05-18 |
2024-04-09 |
Voyager Therapeutics, Inc. |
Compositions and methods for treating Huntington's disease
|
KR20240056729A
(ko)
|
2016-05-18 |
2024-04-30 |
보이저 테라퓨틱스, 인크. |
조절성 폴리뉴클레오티드
|
CA3035522A1
(en)
|
2016-08-30 |
2018-03-08 |
The Regents Of The University Of California |
Methods for biomedical targeting and delivery and devices and systems for practicing the same
|
WO2018094251A1
(en)
|
2016-11-17 |
2018-05-24 |
Kaspar Brian K |
Intrathecal delivery of recombinant adeno-associated virus encoding methyl-cpg binding protein 2
|
WO2018132747A1
(en)
|
2017-01-13 |
2018-07-19 |
University Of Iowa Research Foundation |
Bocaparvovirus small noncoding rna and uses thereof
|
MA47800A
(fr)
|
2017-03-17 |
2020-01-22 |
Univ Newcastle |
Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire
|
CN110997923B
(zh)
|
2017-03-17 |
2024-01-02 |
全国儿童医院研究所 |
腺相关病毒载体递送肌肉特异性微肌营养不良蛋白以治疗肌营养不良症
|
CA3061652A1
(en)
|
2017-05-05 |
2018-11-08 |
Voyager Therapeutics, Inc. |
Compositions and methods of treating amyotrophic lateral sclerosis (als)
|
CN111108198A
(zh)
|
2017-05-05 |
2020-05-05 |
沃雅戈治疗公司 |
治疗亨廷顿病的组合物和方法
|
JOP20190269A1
(ar)
|
2017-06-15 |
2019-11-20 |
Voyager Therapeutics Inc |
بولي نوكليوتيدات aadc لعلاج مرض باركنسون
|
EP3652324A1
(en)
|
2017-07-08 |
2020-05-20 |
Genethon |
Treatment of spinal muscular atrophy
|
AU2018302016A1
(en)
|
2017-07-17 |
2020-02-06 |
The Regents Of The University Of California |
Trajectory array guide system
|
JP7221275B2
(ja)
|
2017-08-03 |
2023-02-13 |
ボイジャー セラピューティクス インコーポレイテッド |
Aavを送達するための組成物および方法
|
US20200237799A1
(en)
|
2017-10-16 |
2020-07-30 |
Voyager Therapeutics, Inc. |
Treatment of amyotrophic lateral sclerosis (als)
|
TWI804518B
(zh)
|
2017-10-16 |
2023-06-11 |
美商航海家醫療公司 |
肌萎縮性脊髓側索硬化症(als)之治療
|
WO2019078916A1
(en)
|
2017-10-18 |
2019-04-25 |
Research Institute At Nationwide Children's Hospital |
ADENO-ASSOCIATED VIRUS VECTOR ADMINISTRATION OF MUSCLE-SPECIFIC MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY
|
KR20200083495A
(ko)
|
2017-10-20 |
2020-07-08 |
더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 |
Nt-3 유전자 치료를 위한 방법 및 물질
|
SG11202012450QA
(en)
|
2018-06-18 |
2021-01-28 |
Res Inst Nationwide Childrens Hospital |
Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
|
WO2019246125A1
(en)
|
2018-06-18 |
2019-12-26 |
Research Institute At Nationwide Children's Hospital |
Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies
|
EP3814514A1
(en)
|
2018-06-29 |
2021-05-05 |
Research Institute at Nationwide Children's Hospital |
Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
|
AU2019328270A1
(en)
|
2018-08-29 |
2021-03-25 |
Research Institute At Nationwide Children's Hospital |
Products and methods for inhibition of expression of mutant GARS protein
|
US11149256B2
(en)
|
2018-09-26 |
2021-10-19 |
California Institute Of Technology |
Adeno-associated virus compositions for targeted gene therapy
|
SG11202103151RA
(en)
*
|
2018-09-28 |
2021-04-29 |
Voyager Therapeutics Inc |
Frataxin expression constructs having engineered promoters and methods of use thereof
|
EP3898995A1
(en)
|
2018-12-21 |
2021-10-27 |
Genethon |
Expression cassettes for gene therapy vectors
|
US20220106592A1
(en)
|
2018-12-31 |
2022-04-07 |
Research Institute At Nationwide Children's Hospital |
DUX4 RNA Silencing Using RNA Targeting CRISPR-CAS13b
|
KR20210124299A
(ko)
|
2019-02-04 |
2021-10-14 |
더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 |
Cln6 폴리뉴클레오티드의 아데노-관련 바이러스 전달
|
BR112021015285A2
(pt)
|
2019-02-04 |
2021-10-05 |
Research Institute At Nationwide Children's Hospital |
Entrega de vírus adenoassociado de polinucleotídeo cln3
|
KR20210133242A
(ko)
|
2019-02-26 |
2021-11-05 |
더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 |
B-사르코글리칸의 아데노 관련 바이러스 벡터 전달 및 근이영양증의 치료
|
US20220193259A1
(en)
|
2019-04-15 |
2022-06-23 |
Sanford Research |
Gene therapy for treating or preventing visual effects in batten disease
|
KR20220009427A
(ko)
|
2019-05-17 |
2022-01-24 |
더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 |
글리코시드 가수분해효소를 사용하는 망막 세포에 대한 유전자 치료 벡터의 개선된 전달
|
KR20220092489A
(ko)
|
2019-08-21 |
2022-07-01 |
더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 |
알파-사코글리칸의 아데노 관련 바이러스 벡터 전달 및 근위축증 치료
|
EP4045662A1
(en)
|
2019-10-18 |
2022-08-24 |
Research Institute at Nationwide Children's Hospital |
Materials and methods for the treatment of disorders associated mutations in the irf2bpl gene
|
EP4045092A1
(en)
|
2019-10-18 |
2022-08-24 |
Research Institute at Nationwide Children's Hospital |
Gene therapy targeting cochlear cells
|
AU2020376048A1
(en)
*
|
2019-11-01 |
2022-06-02 |
Sangamo Therapeutics, Inc. |
Compositions and methods for genome engineering
|
JP2023502474A
(ja)
|
2019-11-22 |
2023-01-24 |
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 |
Ighmbp2遺伝子に関連する障害の治療のための材料および方法
|
CA3165469A1
(en)
|
2019-12-20 |
2021-06-24 |
Research Institute At Nationwide Children's Hospital |
Optimized gene therapy for targeting muscle in muscle diseases
|
IL295698A
(en)
|
2020-02-18 |
2022-10-01 |
Res Inst Nationwide Childrens Hospital |
AAV-mediated targeting of mirna in the treatment of X-linked disorders
|
CA3174863A1
(en)
|
2020-04-14 |
2021-10-21 |
Ana BUJ BELLO |
Vectors for the treatment of acid ceramidase deficiency
|
EP4164668A1
(en)
|
2020-06-15 |
2023-04-19 |
Research Institute at Nationwide Children's Hospital |
Adeno-associated virus vector delivery for muscular dystrophies
|
TW202227634A
(zh)
|
2020-09-08 |
2022-07-16 |
美商薩羅塔治療公司 |
表現γ—肌聚醣之腺相關病毒載體之全身性遞送及肌肉失養症之治療
|
CA3195233A1
(en)
|
2020-09-15 |
2022-03-24 |
Research Institute At Nationwide Children's Hospital |
Aav-mediated homology-independent targeted integration gene editing for correction of diverse dmd mutations in patients with muscular dystrophy
|
EP4217375A1
(en)
|
2020-09-28 |
2023-08-02 |
Research Institute at Nationwide Children's Hospital |
Products and methods for treating muscular dystrophy
|
JP2023548816A
(ja)
|
2020-11-03 |
2023-11-21 |
ファイザー・インク |
陰イオン交換クロマトグラフィーによるaavベクターの精製方法
|
JP2023551279A
(ja)
|
2020-11-30 |
2023-12-07 |
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 |
顔面肩甲上腕筋ジストロフィー(fshd)を治療するための組成物及び方法
|
IL303959A
(en)
|
2020-12-23 |
2023-08-01 |
Pfizer |
Methods for purification of AAV vectors by affinity chromatography
|
WO2022164860A1
(en)
|
2021-01-27 |
2022-08-04 |
Research Institute At Nationwide Children's Hospital |
Materials and methods for the treatment of lysosomal acid lipase deficiency (lal-d)
|
KR20230138949A
(ko)
|
2021-02-03 |
2023-10-05 |
더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 |
Dux4 과발현과 연관된 질환을 치료하기 위한 조성물 및 방법
|
WO2022170038A1
(en)
|
2021-02-05 |
2022-08-11 |
Amicus Therapeutics, Inc. |
Adeno-associated virus delivery of cln3 polynucleotide
|
US11760788B2
(en)
|
2021-03-02 |
2023-09-19 |
Pathways Neuro Pharma, Inc. |
Neuroreceptor compositions and methods of use
|
AU2022229489A1
(en)
|
2021-03-04 |
2023-08-31 |
Research Institute At Nationwide Children's Hospital |
Products and methods for treatment of dystrophin-based myopathies using crispr-cas9 to correct dmd exon duplications
|
WO2022208342A1
(en)
|
2021-04-01 |
2022-10-06 |
Pfizer Inc. |
Pharmaceutical compositions containing adeno-associated viral vector
|
US20240189452A1
(en)
|
2021-04-13 |
2024-06-13 |
Research Institute At Nationwide Children's Hospital |
Recombinant Adeno-Associated Virus Encoding Methyl-CPG Binding Protein 2 for Treating PITT Hopkins Syndrome VIA Intrathecal Delivery
|
EP4323013A2
(en)
|
2021-04-13 |
2024-02-21 |
Capsida, Inc. |
Selected aav compositions having preferred brain enrichment
|
CA3217491A1
(en)
|
2021-04-23 |
2022-10-27 |
Research Institute At Nationwide Children's Hospital |
Products and methods for treating muscular dystrophy
|
CN117377500A
(zh)
|
2021-04-26 |
2024-01-09 |
阿雷克森国际制药有限公司 |
具有改善的组织向性的腺相关病毒载体衣壳
|
JP2024519799A
(ja)
|
2021-05-17 |
2024-05-21 |
サレプタ セラピューティクス, インコーポレイテッド |
筋ジストロフィを処置するための組換えaavベクターの産生
|
EP4108263A3
(en)
|
2021-06-02 |
2023-03-22 |
Research Institute at Nationwide Children's Hospital |
Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
|
KR20240095165A
(ko)
|
2021-08-11 |
2024-06-25 |
솔리드 바이오사이언시즈 인크. |
근이영양증의 치료
|
CA3234702A1
(en)
|
2021-10-07 |
2023-04-13 |
Research Institute At Nationwide Children's Hospital |
Products and methods for myelin protein zero silencing and treating cmt1b disease
|
WO2023060233A1
(en)
|
2021-10-08 |
2023-04-13 |
Amicus Therapeutics, Inc. |
Biomarkers for lysosomal storage diseases
|
US20230139985A1
(en)
|
2021-10-15 |
2023-05-04 |
Research Institute At Nationwide Children's Hospital |
Self-Complementary Adeno-Associated Virus Vector and its Use in Treatment of Muscular Dystrophy
|
EP4186919A1
(en)
|
2021-11-30 |
2023-05-31 |
Research Institute at Nationwide Children's Hospital |
Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
|
EP4198134A1
(en)
|
2021-12-16 |
2023-06-21 |
Genethon |
Gamma-sarcoglycan gene transfer increase using modified itr sequences
|
EP4198046A1
(en)
|
2021-12-16 |
2023-06-21 |
Genethon |
Alpha-sarcoglycan gene transfer increase using modified itr sequences
|
EP4198048A1
(en)
|
2021-12-16 |
2023-06-21 |
Genethon |
Calpain-3 gene transfer increase using modified itr sequences
|
EP4198047A1
(en)
|
2021-12-16 |
2023-06-21 |
Genethon |
Fukutin related protein gene transfer increase using modified itr sequences
|
WO2023122669A1
(en)
|
2021-12-21 |
2023-06-29 |
Research Institute At Nationwide Children's Hospital |
Materials and methods for the treatment of limb girdle muscular dystrophy
|
WO2023168400A2
(en)
|
2022-03-03 |
2023-09-07 |
Research Institute At Nationwide Children's Hospital |
Materials and methods for the treatment of eif2b5 mutations and diseases resulting therefrom
|
WO2023240177A1
(en)
|
2022-06-08 |
2023-12-14 |
Research Instiitute At Nationwide Children's Hospital |
Products and methods for treating diseases or conditions associated with mutant or pathogenic kcnq3 expression
|
WO2024003687A1
(en)
|
2022-06-28 |
2024-01-04 |
Pfizer Inc. |
Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
|
WO2024011115A1
(en)
|
2022-07-06 |
2024-01-11 |
Research Institute At Nationwide Children's Hospital |
Adeno-associated virus delivery of cln1 polynucleotide
|
WO2024035782A1
(en)
|
2022-08-10 |
2024-02-15 |
Aav Gene Therapeutics, Inc. |
Aav-mediated intramuscular delivery of insulin
|
WO2024038365A1
(en)
|
2022-08-16 |
2024-02-22 |
Pfizer Inc. |
Methods for purification of aav vectors by anion exchange chromatography
|
WO2024064913A1
(en)
|
2022-09-23 |
2024-03-28 |
Sarepta Therapeutics, Inc. |
Recombinant aav vectors for treating muscular dystrophy
|
WO2024081706A1
(en)
|
2022-10-11 |
2024-04-18 |
Research Institute At Nationwide Children's Hospital |
Adeno-associated virus delivery to treat spinal muscular atrophy with respiratory distress type 1 (smard1) and charcot-marie-tooth type 2s (cmt2s)
|
WO2024151982A1
(en)
|
2023-01-13 |
2024-07-18 |
Amicus Therapeutics, Inc. |
Gene therapy constructs for the treatment of pompe disease
|