Academic Journal

An optogenetic cell therapy to restore control of target muscles in an aggressive mouse model of amyotrophic lateral sclerosis

التفاصيل البيبلوغرافية
العنوان: An optogenetic cell therapy to restore control of target muscles in an aggressive mouse model of amyotrophic lateral sclerosis
المؤلفون: Bryson, JB, Kourgiantaki, A, Jiang, D, Demosthenous, A, Greensmith, L
المصدر: eLife , 12 , Article RP88250. (2024)
بيانات النشر: eLife Sciences Publications, Ltd
سنة النشر: 2024
المجموعة: University College London: UCL Discovery
مصطلحات موضوعية: ALS, Amyotrophic Lateral Sclerosis, cell transplant, motor neurons, mouse, optogenetics, paralysis, regenerative medicine, stem cells, Animals, Mice, Humans, Muscle, Skeletal, Cell- and Tissue-Based Therapy, Disease Models, Animal
الوصف: Breakdown of neuromuscular junctions (NMJs) is an early pathological hallmark of amyotrophic lateral sclerosis (ALS) that blocks neuromuscular transmission, leading to muscle weakness, paralysis and, ultimately, premature death. Currently, no therapies exist that can prevent progressive motor neuron degeneration, muscle denervation, or paralysis in ALS. Here, we report important advances in the development of an optogenetic, neural replacement strategy that can effectively restore innervation of severely affected skeletal muscles in the aggressive SOD1G93A mouse model of ALS, thus providing an interface to selectively control the function of targeted muscles using optical stimulation. We also identify a specific approach to confer complete survival of allogeneic replacement motor neurons. Furthermore, we demonstrate that an optical stimulation training paradigm can prevent atrophy of reinnervated muscle fibers and results in a tenfold increase in optically evoked contractile force. Together, these advances pave the way for an assistive therapy that could benefit all ALS patients.
نوع الوثيقة: article in journal/newspaper
وصف الملف: application/pdf
اللغة: English
Relation: https://discovery.ucl.ac.uk/id/eprint/10186337/1/elife-88250-v1.pdf; https://discovery.ucl.ac.uk/id/eprint/10186337/
الاتاحة: https://discovery.ucl.ac.uk/id/eprint/10186337/1/elife-88250-v1.pdf
https://discovery.ucl.ac.uk/id/eprint/10186337/
Rights: open
رقم الانضمام: edsbas.3795082A
قاعدة البيانات: BASE