Clicky

Recent developments and industry interest in gene therapy for duchenne muscular dystrophy

13 March 25 Articles ، Toshifumi Yokota Gene therapy for Duchenne muscular dystrophy ، CRISPR-based gene editing techniques ، micro-dystrophin therapy ، AAV vector-based treatment ، gene therapy safety challenges ، FDA approvals for gene therapy
Recent developments and industry interest in gene therapy for duchenne muscular dystrophy
Recent Developments and Industry Interest in Gene Therapy for Duchenne Muscular Dystrophy Introduction Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder that causes progressive muscle degeneration. This condition is caused by mutations in the DMD gene, which encodes a crucial protein for muscle stability. Symptoms usually appear between ages 2 and 5, leading to muscle weakness and, eventually, respiratory or cardiac failure. While antisense oligonucleotides have been approved to …
more

In Silico Screening Based on Predictive Algorithms as a Design Tool for Exon Skipping Oligonucleotides in Duchenne Muscular Dystrophy

06 March 25 Articles ، Toshifumi Yokota ، Yusuke Echigoya ، Vincent Mouly ، Luis Garcia Duchenne Muscular Dystrophy ، Exon skipping ، Duchenne muscular dystrophy ، antisense oligonucleotides ، computational modeling ، efficacy prediction
In Silico Screening Based on Predictive Algorithms as a Design Tool for Exon Skipping Oligonucleotides in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a genetic disorder leading to muscle degeneration. This study introduces a predictive computational model for exon skipping therapy, achieving 89% accuracy for PMOs and 76% accuracy for 2’-O-Methyl RNA, aiding in oligonucleotide selection.
more

Direct Reprogramming of Human DMD Fibroblasts into Myotubes for In Vitro Evaluation of Antisense-Mediated Exon Skipping and Exons 45–55 Skipping Accompanied by Rescue of Dystrophin Expression

15 November 24 Articles ، Toshifumi Yokota Duchenne muscular dystrophy exon skipping ، Dystrophin restoration in DMD ، phosphorodiamidate morpholino oligomers (PMOs) ، Antisense oligonucleotide therapy for DMD ، Direct reprogramming of fibroblasts ، MyoD-induced myotube formation ، Exons 45-55 skipping therapy ، Phosphorodiamidate morpholino oligomers (PMOs) ، Non-invasive DMD drug testing
Direct Reprogramming of Human DMD Fibroblasts into Myotubes for In Vitro Evaluation of Antisense-Mediated Exon Skipping and Exons 45–55 Skipping Accompanied by Rescue of Dystrophin Expression
This study explores direct reprogramming of DMD fibroblasts into myotubes as a non-invasive model for testing antisense oligonucleotide (AO)-mediated exon skipping. Using MyoD gene induction, researchers converted fibroblasts into muscle cells, then applied phosphorodiamidate morpholino oligomers (PMOs) to skip exons 45–55, restoring dystrophin expression. This scalable, reproducible method provides an alternative to muscle biopsies, advancing DMD drug development.
more

Insights from 'Endosomal Escape and Nuclear Localization: Critical Barriers for Antisense Therapeutics

31 October 24 Articles ، Toshifumi Yokota Antisense oligonucleotide therapy ، Nuclear localization in gene therapy ، ASO delivery challenges ، Endosomal escape strategies ، Lipid nanoparticles for ASOs ، Peptide-based ASO delivery ، Non-viral ASO transport ، Gene-targeting therapies
Insights from 'Endosomal Escape and Nuclear Localization: Critical Barriers for Antisense Therapeutics
Antisense oligonucleotides (ASOs) struggle with endosomal entrapment and inefficient nuclear localization, limiting their therapeutic impact. Researchers explore solutions like endosomal escape agents, peptide-based transport, lipid nanoparticles (LNPs), and nuclear localization signals (NLS) to enhance delivery. Combining non-toxic endosomal escape mechanisms with NLS-enhanced strategies could significantly improve ASO therapies, making them more effective for disease treatment.
more

Endosomal Escape and Nuclear Localization: Critical Barriers for Antisense Therapeutics

26 October 24 Articles ، Toshifumi Yokota Antisense oligonucleotide delivery ، ASO endosomal escape ، Nuclear localization in gene therapy ، Splice-switching oligonucleotides (SSOs) ، Non-viral delivery of ASOs ، Lipid nanoparticles for ASO therapy ، Enhancing ASO therapeutic efficiency ، pH-responsive ASO carriers
Endosomal Escape and Nuclear Localization: Critical Barriers for Antisense Therapeutics
Antisense oligonucleotides (ASOs) are promising gene-targeting therapies, but endosomal escape and nuclear localization limit their effectiveness. Only 1-2% of ASOs reach their target mRNA. Strategies like lipid nanoparticles, pH-responsive materials, and membrane-destabilizing agents aim to improve ASO delivery. Enhancing nuclear localization is also critical for splice-switching oligonucleotides (SSOs). Overcoming these barriers is essential for making ASO-based therapies viable in clinical settings.
more

Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and Challenges

16 October 24 Articles ، Toshifumi Yokota ، Akinori Nakamura ، Yusuke Echigoya multiple exon skipping Duchenne muscular dystrophy ، exon 45-55 skipping therapy ، gene therapy for DMD ، antisense oligonucleotides for Duchenne muscular dystrophy ، dystrophin restoration therapy ، phosphorodiamidate morpholino oligomers (PMOs) ، vivo-PMOs for DMD ، clinical trials for multiple exon skipping ، Duchenne muscular dystrophy gene editing ، DMD exon-skipping hot spots
Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and Challenges
Multiple exon skipping is a promising therapy for Duchenne Muscular Dystrophy (DMD), targeting genetic hot spots like exons 45–55 and 3–9 to restore dystrophin. While PMOs have shown potential, challenges in delivery remain. Advances like vivo-PMOs aim to enhance uptake. Despite promising preclinical results, clinical validation is needed to ensure effectiveness and long-term safety. Ongoing research focuses on optimizing delivery and broadening patient eligibility for exon-skipping therapies.
more

Casimersen for Duchenne muscular dystrophy

15 October 24 Articles ، Toshifumi Yokota Casimersen for Duchenne muscular dystrophy ، Amondys 45 FDA approval ، Exon 45 skipping therapy ، ESSENCE Phase III trial ، Dystrophin restoration in DMD ، Gene therapy for Duchenne muscular dystrophy ، Casimersen side effects ، Long-term efficacy of exon skipping therapies ، DMD clinical trials update ، Casimersen intravenous treatment
Casimersen for Duchenne muscular dystrophy
Casimersen (Amondys 45) is an FDA-approved exon 45 skipping therapy for Duchenne Muscular Dystrophy (DMD). Learn about its efficacy, side effects, and ongoing ESSENCE Phase III trial.
more

Recent Progress of Antisense Oligonucleotide Therapy for Superoxide Dismutase 1-mutated Amyotrophic Lateral Sclerosis: Focus on Tofersen

12 October 24 Articles ، Toshifumi Yokota Antisense oligonucleotide therapy for ALS ، Tofersen FDA approval ، SOD1 mutation in ALS ، ALS genetic treatments ، Intrathecal injection ALS treatment ، Neurodegenerative disease therapies ، ASO therapy in neurodegeneration ، Tofersen clinical trials ، ALS biomarkers and treatment ، Future of antisense oligonucleotide therapy
Recent Progress of Antisense Oligonucleotide Therapy for Superoxide Dismutase 1-mutated Amyotrophic Lateral Sclerosis: Focus on Tofersen
Tofersen is an FDA-approved antisense oligonucleotide therapy targeting SOD1 mutations in ALS. This treatment reduces toxic SOD1 protein levels, showing promise in slowing disease progression. Despite challenges with intrathecal administration, ongoing research explores improved delivery methods and earlier interventions for presymptomatic carriers.
more

Antisense and Gene Therapy Options for Duchenne Muscular Dystrophy Arising from Mutations in the N-Terminal Hotspot

17 September 24 Articles ، Toshifumi Yokota Antisense therapy for DMD mutations ، Gene therapy in Duchenne muscular dystrophy ، Exon skipping treatments for DMD ، AAV vectors in gene therapy for DMD ، Microdystrophin therapy for Duchenne patients ، Targeting exon 2–22 mutations in DMD ، N-terminal hotspot mutations in dystrophin gene
Antisense and Gene Therapy Options for Duchenne Muscular Dystrophy Arising from Mutations in the N-Terminal Hotspot
This article reviews antisense and gene therapy strategies for DMD mutations in the exon 2–22 region, focusing on exon skipping, AAV vectors, and microdystrophin therapy.
more

Oligonucleotide Therapies for Facioscapulohumeral Muscular Dystrophy: Current Preclinical Landscape

29 August 24 Articles ، Toshifumi Yokota Oligonucleotide therapies for FSHD ، DUX4-targeting treatments in muscular dystrophy ، RNA interference for FSHD therapy ، Antisense oligonucleotides in FSHD research ، CRISPR-based gene silencing in FSHD ، Epigenetic therapies for muscular dystrophy ، D4Z4 array repression in FSHD
Oligonucleotide Therapies for Facioscapulohumeral Muscular Dystrophy: Current Preclinical Landscape
This article reviews oligonucleotide-based therapies for facioscapulohumeral muscular dystrophy, highlighting antisense oligonucleotides, RNAi, and CRISPR-based approaches targeting DUX4.
more

Recent Progress in Gene-Targeting Therapies for Spinal Muscular Atrophy: Promises and Challenges

10 August 24 Articles ، Toshifumi Yokota Gene therapy for spinal muscular atrophy ، SMA gene-targeting treatments ، Antisense oligonucleotide therapy for SMA ، Onasemnogene abeparvovec gene replacement ، Risdiplam small molecule treatment ، Challenges in SMA therapies ، SMN1 and SMN2 gene therapy
Recent Progress in Gene-Targeting Therapies for Spinal Muscular Atrophy: Promises and Challenges
This review discusses gene-targeting therapies for spinal muscular atrophy, covering antisense oligonucleotides, gene replacement, small molecules, and treatment challenges.
more

The Dysferlinopathies Conundrum: Clinical Spectra, Disease Mechanism and Genetic Approaches for Treatments

15 May 24 Articles ، Toshifumi Yokota Dysferlinopathies clinical features ، DYSF gene mutations and muscle weakness ، Muscle membrane repair disorders ، Genetic therapies for dysferlinopathies ، Pharmacological treatments for DYSF-related diseases ، Precision medicine in muscular dystrophies ، Animal models for dysferlinopathy research
The Dysferlinopathies Conundrum: Clinical Spectra, Disease Mechanism and Genetic Approaches for Treatments
This review examines dysferlinopathies, highlighting clinical features, molecular mechanisms, genetic therapies, and the latest research in treatment development.
more

Duchenne muscular dystrophy: promising early-stage clinical trials to watch

21 March 24 Articles ، Toshifumi Yokota Duchenne muscular dystrophy clinical trials ، Early-stage DMD treatments ، Exon-skipping therapies for DMD ، Gene therapy for Duchenne dystrophy ، Micro-dystrophin gene therapy ، AON treatments for DMD ، New drugs for Duchenne muscular dystrophy
Duchenne muscular dystrophy: promising early-stage clinical trials to watch
This review highlights early-stage clinical trials for Duchenne muscular dystrophy, covering exon-skipping therapies, gene therapy, and novel treatment strategies.
more

Recent Posts

Categories

I'd be delighted if you could explore the other sections of my website.

Biochemist Researcher . YouTuber . Medical Laboratory Tech

!I am Ali Nik Akhtar

Personal Website​​​​​​​

If you have any questions or would like to discuss further, please feel free to email me. I would be delighted to get to know you better.

Ready to start a collaboration...​​​​​​​

Contact Me

Nikakhtar422@gmail.com

All rights reserved. This website belongs to Ali Nik Akhtar.