Results 71 to 80 of about 2,159 (173)

New Therapeutics Options for Pediatric Neuromuscular Disorders

Frontiers in Pediatrics, 2020
Neuromuscular disorders (NMDs) of Childhood onset are a genetically heterogeneous group of diseases affecting the anterior horn cell, the peripheral nerve, the neuromuscular junction, or the muscle.
Marina Flotats-Bastardas, Andreas Hahn
doaj   +1 more source

Delivery Systems for Therapeutic Genome Editing: Challenges, Innovations, and Future Perspectives

MedComm, Volume 7, Issue 7, July 2026.
Schematic illustration of four emerging CRISPR–Cas delivery platforms defined by distinct design principles and structural features: virus‐mimicking nanosystems (e.g., VLPs), cell‐derived extracellular vesicles, cell‐penetrating peptides, and stimuli‐responsive scaffolds. These platforms enable spatiotemporally controlled delivery of RNPs, mRNA, or DNA
Meijia Yang, Yiqiong Song, Ziyang Wang, Ke Chao, Lifeng Li, Xu Zhang, Xiaoran Duan, Chenglong Yu, Ruyue Xue, Jie Zhao   +9 more
wiley   +1 more source

Improved therapeutic approach for spinal muscular atrophy via ubiquitination‐resistant survival motor neuron variant

Journal of Cachexia, Sarcopenia and Muscle
Background Zolgensma is a gene‐replacement therapy that has led to a promising treatment for spinal muscular atrophy (SMA). However, clinical trials of Zolgensma have raised two major concerns: insufficient therapeutic effects and adverse events.
Joonwoo Rhee, Jong‐Seol Kang, Young‐Woo Jo, Kyusang Yoo, Ye Lynne Kim, Sang‐Hyeon Hann, Yea‐Eun Kim, Hyun Kim, Ji‐Hoon Kim, Young‐Yun Kong   +9 more
doaj   +1 more source

Development of an Agent‐Based Model to Investigate Durability of Factor IX Activity in Hemophilia B Patients Treated With Etranacogene Dezaparvovec

CPT: Pharmacometrics &Systems Pharmacology, Volume 15, Issue 7, July 2026.
ABSTRACT Many currently approved gene therapies use adeno‐associated virus (AAV) to deliver DNA sequences encoding protein(s)‐of‐interest into cells. The AAV viral genome forms stable, circular DNA structures called episomes after entering the nuclei. Therapeutic proteins are then generated in vivo from transcription and translation of these episomes ...
Yuezhe Li, Partha Nandy, Eric Jordie, Timothy Knab, Karsten Peppel, Daniel C. Kirouac, A. Katharina Wilkins, Silpa Nuthalapati   +7 more
wiley   +1 more source

From Nexavar to Zolgensma: Has Indian Compulsory Licensing Law Evolved?

The compulsory licence issued in India in 2012 for Bayer's cancer drug Nexavar remains the seminal instance of section 84 of the Patents Act, 1970 in action. The case is frequently read in India either as proof that patient health trumps patent rights or
Ujjawal Solanki
core   +1 more source

AAV-aMTD-Parkin, a therapeutic gene delivery cargo, enhances motor and cognitive functions in Parkinson's and Alzheimer’s diseases

Pharmacological Research
Neurodegenerative disorders, such as Parkinson's disease (PD) and Alzheimer's disease (AD), have a global prevalence and profoundly impact both motor and cognitive functions. Although adeno-associated virus (AAV)-based gene therapy has shown promise, its
Seokwon Lee, Mingu Kang, Seungwoo Lee, Sangsun Yoon, Yeonjin Cho, Dongjae Min, Daye Ann, Jisoo Shin, Young-Ki Paik, Daewoong Jo   +9 more
doaj   +1 more source

Fortalecimento de capacidades estatais: estudo de caso sobre a atuação dos NATJUS na incorporação do Zolgensma para o tratamento da Atrofia Muscular Espinhal Tipo 1 [PDF]

, 2023
Por meio deste estudo de caso da atuação dos Núcleos de Apoio Técnico ao Poder Judiciário – NATJUS, vinculados aos Tribunais de Justiça de São Paulo e do Distrito Federal e Territórios, lança-se luz às potencialidades advindas da aplicação dos marcos ...
Dias Júnior, Osmar Sebastião
core  

Real-World Data in Children with Spinal Muscular Atrophy Type 1 on Long-Term Ventilation Receiving Gene Therapy: A Prospective Cohort Study

Advances in Respiratory Medicine
Patients with spinal muscular atrophy type 1 (SMA-1) requiring invasive ventilation can be eligible for gene therapy if they tolerate at least 8 h off ventilation per day.
Mohammad Ala’ Alajjuri, Rania Abusamra, Vivek Mundada, Omendra Narayan   +3 more
doaj   +1 more source

Engineering Targeted Gene Delivery Systems for Primary Hereditary Skeletal Myopathies: Current Strategies and Future Perspectives

Biomedicines
Skeletal muscle, constituting ~40% of body mass, serves as a primary effector for movement and a key metabolic regulator through myokine secretion. Hereditary myopathies, including dystrophinopathies (DMD/BMD), limb–girdle muscular dystrophies (LGMD ...
Jiahao Wu, Yimin Hua, Yanjiang Zheng, Xu Liu, Yifei Li   +4 more
doaj   +1 more source

Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor

EMBO Molecular Medicine
Physiological regulation of transgene expression is a major challenge in gene therapy. Onasemnogene abeparvovec (Zolgensma®) is an approved adeno-associated virus (AAV) vector gene therapy for infants with spinal muscular atrophy (SMA), however, adverse ...
Qing Xie, Xiupeng Chen, Hong Ma, Yunxiang Zhu, Yijie Ma, Leila Jalinous, Gerald F Cox, Fiona Weaver, Jun Yang, Zachary Kennedy, Alisha Gruntman, Ailing Du, Qin Su, Ran He, Phillip WL Tai, Guangping Gao, Jun Xie   +16 more
doaj   +1 more source