標(biāo)題: Titlebook: Exon Skipping and Inclusion Therapies; Methods and Protocol Toshifumi Yokota,Rika Maruyama Book 2018 Springer Science+Business Media, LLC, [打印本頁] 作者: 短暫 時間: 2025-3-21 18:03
書目名稱Exon Skipping and Inclusion Therapies影響因子(影響力)
書目名稱Exon Skipping and Inclusion Therapies影響因子(影響力)學(xué)科排名
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書目名稱Exon Skipping and Inclusion Therapies被引頻次
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書目名稱Exon Skipping and Inclusion Therapies讀者反饋
書目名稱Exon Skipping and Inclusion Therapies讀者反饋學(xué)科排名
作者: COWER 時間: 2025-3-21 21:05 作者: 恃強(qiáng)凌弱 時間: 2025-3-22 00:29 作者: 短程旅游 時間: 2025-3-22 08:04
https://doi.org/10.1007/978-3-031-14406-6ase. Defects in RNA splicing account for at least 10% of all genetic disorders, with the number expected to increase as more information is uncovered on the contribution of noncoding genomic regions to disease. Splice modulation through the use of antisense oligonucleotides (AOs) has emerged as a pr作者: MUT 時間: 2025-3-22 11:45
Giordano Zambelli,Luciano Morgantiproach uses antisense oligonucleotides (AON) to modify the splicing of pre-mRNA to correct the mutation responsible for a disease, or to suppress a particular gene expression, as in allergic diseases. Antisense-mediated exon skipping is most extensively studied in Duchenne muscular dystrophy (DMD) a作者: multiply 時間: 2025-3-22 15:05
Ultrasound, Embodiment and Abortion,tein. Insufficient levels of SMN results in the loss of motor neurons, which causes muscle weakness, respiratory distress, and paralysis. A nearly identical gene (.) contains a C-to-T transition which excludes exon 7 from 90% of the mature mRNA transcripts, leading to unstable proteins which are tar作者: multiply 時間: 2025-3-22 21:08 作者: mercenary 時間: 2025-3-22 23:24 作者: 創(chuàng)新 時間: 2025-3-23 01:25 作者: Critical 時間: 2025-3-23 07:06 作者: Senescent 時間: 2025-3-23 13:12 作者: CROAK 時間: 2025-3-23 15:47
J. Nagarjun,S. Manimaran,M. KrishnaprakashDuchenne muscular dystrophy (DMD). Systemic administration of antisense phosphorodiamidate morpholino oligomers (PMOs) targeting exons 6 and 8 in dystrophin mRNA of the canine X-linked muscular dystrophy model in Japan (CXMD.) that lacks exon 7, restored dystrophin expression throughout skeletal mus作者: travail 時間: 2025-3-23 20:19 作者: Root494 時間: 2025-3-23 23:23 作者: 脫毛 時間: 2025-3-24 02:39
https://doi.org/10.1007/978-3-540-88087-5for myofiber integrity. Exon skipping therapy is an emerging strategy for restoring the open reading frame of the . gene to produce functional protein in DMD patients by skipping single or multiple exons. Although antisense oligonucleotides are able to target pre-mRNA for exon skipping, their half-l作者: hedonic 時間: 2025-3-24 07:26 作者: 勾引 時間: 2025-3-24 10:59
Fuzziness in Information Systemsinvolves the following two aspects: (1) efficiency and accuracy of exon skipping and levels of dystrophin expression determined by RT-PCR, immunochemistry, and western blotting; (2) therapeutic effects on muscle pathology and functions assessed by histology and functional assays including grip stren作者: Tracheotomy 時間: 2025-3-24 18:13
Studies in Systems, Decision and Control protein. Antisense oligonucleotide (AON)-mediated exon skipping has been developed as a method to restore the reading frame, which allows the synthesis of internally truncated, but partially functional dystrophin proteins, as found in the less severe Becker muscular dystrophy (BMD). This approach i作者: 先兆 時間: 2025-3-24 20:09
Toshifumi Yokota,Rika MaruyamaIncludes cutting-edge methods and protocols.Provides step-by-step detail essential for reproducible results.Contains key notes and implementation advice from the experts作者: 嚴(yán)重傷害 時間: 2025-3-25 00:21 作者: Nuance 時間: 2025-3-25 05:29 作者: Fulminate 時間: 2025-3-25 08:10 作者: 關(guān)心 時間: 2025-3-25 12:22 作者: GAVEL 時間: 2025-3-25 16:27
Book 2018ble laboratory protocols, and tips on troubleshooting and avoiding known pitfalls...Authoritative and cutting-edge, .Exon Skipping and Inclusion Therapies: Methods and Protocols .aims to help researchers initiate the development of next-generation therapies. .作者: 轎車 時間: 2025-3-25 21:55 作者: laxative 時間: 2025-3-26 02:21 作者: 可觸知 時間: 2025-3-26 05:31 作者: Conquest 時間: 2025-3-26 08:57
Interlocking Socio-Technical Worlds, Food and Drug Agency (FDA) in December?of 2016. Here we briefly review the pharmacological relevance of the drug, clinical trials, toxicity, and future directions following the approval of nusinersen.作者: 陳腐思想 時間: 2025-3-26 13:20 作者: ethereal 時間: 2025-3-26 18:05
Nusinersen in the Treatment of Spinal Muscular Atrophy Food and Drug Agency (FDA) in December?of 2016. Here we briefly review the pharmacological relevance of the drug, clinical trials, toxicity, and future directions following the approval of nusinersen.作者: Alcove 時間: 2025-3-26 21:31 作者: 膠水 時間: 2025-3-27 04:50 作者: grenade 時間: 2025-3-27 06:37
An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for proach uses antisense oligonucleotides (AON) to modify the splicing of pre-mRNA to correct the mutation responsible for a disease, or to suppress a particular gene expression, as in allergic diseases. Antisense-mediated exon skipping is most extensively studied in Duchenne muscular dystrophy (DMD) a作者: Dysarthria 時間: 2025-3-27 12:07
Recent Advances and Clinical Applications of Exon Inclusion for Spinal Muscular Atrophytein. Insufficient levels of SMN results in the loss of motor neurons, which causes muscle weakness, respiratory distress, and paralysis. A nearly identical gene (.) contains a C-to-T transition which excludes exon 7 from 90% of the mature mRNA transcripts, leading to unstable proteins which are tar作者: 慢慢啃 時間: 2025-3-27 13:52
Nusinersen in the Treatment of Spinal Muscular Atrophyss of motor neurons. With the discovery of the intronic splicing silencer N1 (ISS-N1) as a potential target for antisense therapy, several antisense oligonucleotides (ASOs) are being developed to include exon 7 in the final mRNA transcript of the . gene and thereby increasing the production of spina作者: Integrate 時間: 2025-3-27 18:28 作者: 健壯 時間: 2025-3-28 01:49 作者: 注射器 時間: 2025-3-28 03:58 作者: 保守 時間: 2025-3-28 07:32 作者: Morbid 時間: 2025-3-28 10:48 作者: Gudgeon 時間: 2025-3-28 16:53 作者: Insatiable 時間: 2025-3-28 22:13 作者: Allergic 時間: 2025-3-29 02:21
Restoration of Dystrophin Protein Expression by Exon Skipping Utilizing CRISPR-Cas9 in Myoblasts Derfor myofiber integrity. Exon skipping therapy is an emerging strategy for restoring the open reading frame of the . gene to produce functional protein in DMD patients by skipping single or multiple exons. Although antisense oligonucleotides are able to target pre-mRNA for exon skipping, their half-l作者: 抑制 時間: 2025-3-29 04:29 作者: detach 時間: 2025-3-29 09:22
In Vivo Evaluation of Dystrophin Exon Skipping in , Miceinvolves the following two aspects: (1) efficiency and accuracy of exon skipping and levels of dystrophin expression determined by RT-PCR, immunochemistry, and western blotting; (2) therapeutic effects on muscle pathology and functions assessed by histology and functional assays including grip stren作者: Urgency 時間: 2025-3-29 15:08
Exon 51 Skipping Quantification by Digital Droplet PCR in del52hDMD/, Mice protein. Antisense oligonucleotide (AON)-mediated exon skipping has been developed as a method to restore the reading frame, which allows the synthesis of internally truncated, but partially functional dystrophin proteins, as found in the less severe Becker muscular dystrophy (BMD). This approach i作者: dowagers-hump 時間: 2025-3-29 17:54 作者: CAPE 時間: 2025-3-29 20:20
https://doi.org/10.1007/978-3-031-14406-6ations, and its evolution into the approach we are now familiar with. We give a more extensive history of exon skipping in particular, as it is the splice modulation approach given the most focus in this book.作者: CHECK 時間: 2025-3-30 00:55
Giordano Zambelli,Luciano Morganting authorization by the US Food and Drug Administration (FDA), on the condition that additional postapproval trials show clinical benefit. Permanent exon skipping achieved at the DNA level using clustered regularly interspaced short palindromic repeats (CRISPR) technology holds promise in current pr作者: 祝賀 時間: 2025-3-30 07:47 作者: homocysteine 時間: 2025-3-30 08:32 作者: 豎琴 時間: 2025-3-30 14:17 作者: FOLD 時間: 2025-3-30 18:12 作者: 忍耐 時間: 2025-3-30 21:21 作者: single 時間: 2025-3-31 03:51
J. Nagarjun,S. Manimaran,M. Krishnaprakash patient to myotubes by MyoD transduction using fluorescence-activated cell sorting (FACS). We subsequently designed antisense PMOs targeting identical regions of dog and human dystrophin exons 6 and 8 and administered them as a cocktail to the in vitro generated dog or human myotubes. In both cases作者: 全國性 時間: 2025-3-31 07:36
https://doi.org/10.1007/978-3-540-88087-5tiated myoblasts. Herein, we describe an optimized methodology to prepare myoblasts differentiated from iPS cells by mRNA transfection of the CRISPR-Cas9 system to skip exon 45 in myoblasts, and evaluate the restored dystrophin by RT-PCR and Western blotting.作者: canvass 時間: 2025-3-31 09:56 作者: jocular 時間: 2025-3-31 16:13 作者: Liberate 時間: 2025-3-31 19:11 作者: 智力高 時間: 2025-4-1 00:29