Systemic delivery of an AAV9 exonskipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse

05 January 25 Articles ، Kevin Flanigan ، Robert Weiss DMD ، GeneTherapy ، ExonSkipping ، دیستروفی_عضلانی_دوشن ، حذف_اگزون ، ژن_درمانی ، DystrophinRestoration ، بازسازی_دیستروفین ، Dup2MouseModel ، AAV9 ، U7snRNA ، مدل_موش_Dup2
Systemic delivery of an AAV9 exonskipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse
  Background: Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by mutations in the DMD gene. These mutations disrupt the dystrophin protein, essential for muscle function. Current treatments, like exon-skipping oligonucleotides, have limitations due to their short lifespan and need for frequent administration.   Objective: This study explored the use of a self-complementary adeno-associated virus (scAAV9) vector expressing U7 small nuclear RNA (snRNA) to target exon 2 of …
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Challenges of Assessing Exon 53 Skipping of the HumanDMD Transcript with Locked Nucleic Acid-ModifiedAntisense Oligonucleotides in a Mouse Modelfor Duchenne Muscular Dystrophy

02 January 25 Articles ، Annemieke Aartsma-Rus ، Anastasia Khvorova ، Masad J. Damha DMD ، ExonSkipping ، AntisenseTherapy ، حذف_اگزون ، DystrophinRestoration ، بازسازی_دیستروفین ، LNA ، PreclinicalStudy ، دیستروفی_دوشن ، الیگونوکلئوتید
Challenges of Assessing Exon 53 Skipping of the HumanDMD Transcript with Locked Nucleic Acid-ModifiedAntisense Oligonucleotides in a Mouse Modelfor Duchenne Muscular Dystrophy
  Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by mutations in the dystrophin gene, leading to muscle degeneration and early death. Antisense oligonucleotides (AONs) are promising therapeutic agents designed to skip faulty exons in the dystrophin gene and restore functional protein.   This study evaluated the efficiency of AONs targeting exon 53 of the dystrophin gene in a mouse model, using chemically modified AONs to enhance skipping …
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Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy

28 December 24 Articles ، Annemieke Aartsma-Rus ، Judith van Deutekom DuchenneMuscularDystrophy ، ExonSkipping ، دیستروفی_عضلانی_دوشن ، antisenseoligonucleotide ، پرش_اگزون ، AntisenseOligonucleotide ، DystrophinRestoration ، NextGenerationTherapies ، PreclinicalResearch ، الیگونوکلئوتیدآنتی_سنس ، بازگردانی_دیستروفین ، تحقیقات_پیش_بالینی
Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy
Antisense oligonucleotides (AONs) have emerged as a promising therapy for Duchenne Muscular Dystrophy (DMD), a severe neuromuscular disease caused by dystrophin deficiency. Early AON drugs like drisapersen and eteplirsen achieved limited success, leading researchers to develop more efficient next-generation AONs. This study focused on refining AONs for exon 51 skipping through advanced chemical modifications and identifying optimal target sites to enhance efficacy and safety.   Researchers screened over 100 AONs …
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Exon skipping and dystrophin restoration in patientswith Duchenne muscular dystrophy after systemicphosphorodiamidate morpholino oligomer treatment:an open-label, phase 2, dose-escalation study

17 November 24 Articles ، Volker Straub ، Ryszard Kole ، Steve Wilton ، George Dickson ، Karen Anthony ، Francesco Muntoni ، Virginia Arechavala-Gomeza ، Kate Bushby ، Jennifer Morgan DuchenneMuscularDystrophy ، GeneTherapy ، ExonSkipping ، دیستروفی_عضلانی_دوشن ، پرش_اگزون ، DystrophinRestoration ، AVI4658 ، PMO ، ژن تراپی ، بازیابی_دیستروفین
Exon skipping and dystrophin restoration in patientswith Duchenne muscular dystrophy after systemicphosphorodiamidate morpholino oligomer treatment:an open-label, phase 2, dose-escalation study
This study explored the effects of AVI-4658, a phosphorodiamidate morpholino oligomer (PMO), on exon skipping and dystrophin restoration in Duchenne muscular dystrophy (DMD) patients. The open-label, phase 2, dose-escalation trial enrolled 19 boys aged 5-15, treated with doses ranging from 0.5 to 20 mg/kg weekly for 12 weeks. The main aim was to evaluate safety, with secondary goals to assess pharmacokinetics and the efficacy of exon 51 skipping and dystrophin …
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Dystrophin Restoration after Adeno-Associated Virus U7eMediated Dmd Exon Skipping Is Modulated by Muscular Exercise in the Severe D2-Mdx Duchenne Muscular Dystrophy Murine Model

17 November 24 Articles ، Capucine Trollet ، Arnaud Ferry DMD ، DuchenneMuscularDystrophy ، GeneTherapy ، ExonSkipping ، دیستروفی_عضلانی_دوشن ، AAV ، حذف_اگزون ، ژن_درمانی ، VoluntaryExercise ، MuscleDamage ، DystrophinRestoration ، آسیب_عضلانی ، بازسازی_دیستروفین
Dystrophin Restoration after Adeno-Associated Virus U7eMediated Dmd Exon Skipping Is Modulated by Muscular Exercise in the Severe D2-Mdx Duchenne Muscular Dystrophy Murine Model
  This study explores the impact of voluntary exercise on the effectiveness of a gene therapy approach for Duchenne muscular dystrophy (DMD) using the D2-mdx murine model. DMD is caused by mutations in the dystrophin gene, leading to muscle damage and impaired regeneration. The research uses adeno-associated virus (AAV)-mediated U7 snRNA to skip a mutation-containing exon and restore dystrophin production. D2-mdx mice were treated with AAV-U7 and allowed to run …
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