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Lipid nanoparticles and transcranial focused ultrasound enhance the delivery of SOD1 antisense oligonucleotides to the murine brain for ALS therapy.

Autor: Ediriweera GR; Centre for Advanced Imaging and Australian Institute for Bioengineering & Nanotechnology (AIBN), The University of Queensland, Brisbane, QLD 4072, Australia., Sivaram AJ; Centre for Advanced Imaging and Australian Institute for Bioengineering & Nanotechnology (AIBN), The University of Queensland, Brisbane, QLD 4072, Australia., Cowin G; Centre for Advanced Imaging and Australian Institute for Bioengineering & Nanotechnology (AIBN), The University of Queensland, Brisbane, QLD 4072, Australia; National Imaging Facility, Centre for Advanced Imaging, The University of Queensland, Brisbane, QLD 4072, Australia., Brown ML; School of Chemistry and Molecular Bioscience, Molecular Horizons, Faculty of Science, Medicine and Health, University of Wollongong, Wollongong, NSW 2522, Australia., McAlary L; School of Chemistry and Molecular Bioscience, Molecular Horizons, Faculty of Science, Medicine and Health, University of Wollongong, Wollongong, NSW 2522, Australia., Lum JS; School of Chemistry and Molecular Bioscience, Molecular Horizons, Faculty of Science, Medicine and Health, University of Wollongong, Wollongong, NSW 2522, Australia; School of Medical, Indigenous and Health Sciences, Molecular Horizons, Faculty of Science, Medicine and Health, University of Wollongong, Wollongong, NSW 2522, Australia., Fletcher NL; Centre for Advanced Imaging and Australian Institute for Bioengineering & Nanotechnology (AIBN), The University of Queensland, Brisbane, QLD 4072, Australia., Robinson L; School of Chemistry and Molecular Bioscience, Molecular Horizons, Faculty of Science, Medicine and Health, University of Wollongong, Wollongong, NSW 2522, Australia., Simpson JD; Louvain Institute of Biomolecular Science and Technology, Université Catholique de Louvain, 1348 Louvain-la-Neuve, Belgium., Chen L; Queensland Brain Institute (QBI), The University of Queensland, Brisbane, QLD 4072, Australia., Wasielewska JM; Brain and Mental Health Program, QIMR Berghofer Medical Research Institute, Herston, QLD 4006, Australia; Faculty of Medicine, The University of Queensland, Brisbane, QLD 4072, Australia., Byrne E; School of Chemistry and Molecular Bioscience, Molecular Horizons, Faculty of Science, Medicine and Health, University of Wollongong, Wollongong, NSW 2522, Australia., Finnie JW; Faculty of Health and Medical Sciences, School of Biomedicine, University of Adelaide, Adelaide, SA 5000, Australia., Manavis J; Faculty of Health and Medical Sciences, School of Biomedicine, University of Adelaide, Adelaide, SA 5000, Australia., White AR; Brain and Mental Health Program, QIMR Berghofer Medical Research Institute, Herston, QLD 4006, Australia; Faculty of Medicine, The University of Queensland, Brisbane, QLD 4072, Australia., Yerbury JJ; School of Chemistry and Molecular Bioscience, Molecular Horizons, Faculty of Science, Medicine and Health, University of Wollongong, Wollongong, NSW 2522, Australia., Thurecht KJ; Centre for Advanced Imaging and Australian Institute for Bioengineering & Nanotechnology (AIBN), The University of Queensland, Brisbane, QLD 4072, Australia., Vine KL; School of Chemistry and Molecular Bioscience, Molecular Horizons, Faculty of Science, Medicine and Health, University of Wollongong, Wollongong, NSW 2522, Australia. Electronic address: kara@uow.edu.au.

Publikováno v: Journal of controlled release : official journal of the Controlled Release Society [J Control Release] 2024 Dec 12; Vol. 378, pp. 221-235. Date of Electronic Publication: 2024 Dec 12.

Characteristics of Sensory Neuron Dysfunction in Amyotrophic Lateral Sclerosis (ALS): Potential for ALS Therapy

Autor: Soju Seki, Yoshihiro Kitaoka, Sou Kawata, Akira Nishiura, Toshihiro Uchihashi, Shin-ichiro Hiraoka, Yusuke Yokota, Emiko Tanaka Isomura, Mikihiko Kogo, Susumu Tanaka

Publikováno v: Biomedicines, Vol 11, Iss 11, p 2967 (2023)

Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disorder characterised by the progressive degeneration of motor neurons, resulting in muscle weakness, paralysis, and, ultimately, death. Presently, no effective treatment for ALS

Externí odkaz: https://doaj.org/article/28b431bcd21e4a49a347dba8a626ad86

Characteristics of Sensory Neuron Dysfunction in Amyotrophic Lateral Sclerosis (ALS): Potential for ALS Therapy.

Autor: Seki S; Department of Oral and Maxillofacial Surgery, Graduate School of Dentistry, Osaka University, 1-8 Yamadaoka, Suita 565-0871, Osaka, Japan., Kitaoka Y; Department of Oral and Maxillofacial Surgery, Graduate School of Dentistry, Osaka University, 1-8 Yamadaoka, Suita 565-0871, Osaka, Japan., Kawata S; Department of Oral and Maxillofacial Surgery, Graduate School of Dentistry, Osaka University, 1-8 Yamadaoka, Suita 565-0871, Osaka, Japan., Nishiura A; Department of Oral and Maxillofacial Surgery, Graduate School of Dentistry, Osaka University, 1-8 Yamadaoka, Suita 565-0871, Osaka, Japan., Uchihashi T; Department of Oral and Maxillofacial Surgery, Graduate School of Dentistry, Osaka University, 1-8 Yamadaoka, Suita 565-0871, Osaka, Japan., Hiraoka SI; Department of Oral and Maxillofacial Surgery, Graduate School of Dentistry, Osaka University, 1-8 Yamadaoka, Suita 565-0871, Osaka, Japan., Yokota Y; Department of Oral and Maxillofacial Surgery, Graduate School of Dentistry, Osaka University, 1-8 Yamadaoka, Suita 565-0871, Osaka, Japan., Isomura ET; Department of Oral and Maxillofacial Surgery, Graduate School of Dentistry, Osaka University, 1-8 Yamadaoka, Suita 565-0871, Osaka, Japan., Kogo M; Department of Oral and Maxillofacial Surgery, Graduate School of Dentistry, Osaka University, 1-8 Yamadaoka, Suita 565-0871, Osaka, Japan., Tanaka S; Department of Oral and Maxillofacial Surgery, Graduate School of Dentistry, Osaka University, 1-8 Yamadaoka, Suita 565-0871, Osaka, Japan.

Publikováno v: Biomedicines [Biomedicines] 2023 Nov 03; Vol. 11 (11). Date of Electronic Publication: 2023 Nov 03.

Amylyx's ALS therapy secures FDA approval, as regulatory flexibility trumps underwhelming data.

Autor: Mullard A

Publikováno v: Nature reviews. Drug discovery [Nat Rev Drug Discov] 2022 Nov; Vol. 21 (11), pp. 786.