Neurodégénérescences générales - page 2 - Publications Scientifiques

Dernière publication 09/03/2021

Squalenoyl siRNA PMP22 nanoparticles are effective in treating mouse models of C

Charcot-Marie-Tooth disease type 1 A (CMT1A) lacks an effective treatment. We provide a therapy for CMT1A, based on siRNA conjugated to squalene...

Array
(
    [id_prestablog_news] => 1385
    [id_shop] => 1
    [date] => 2021-03-09 00:00:00
    [date_modification] => 2024-02-09 14:15:12
    [langues] => ["1","2"]
    [actif] => 1
    [slide] => 0
    [url_redirect] => 
    [average_rating] => 
    [number_rating] => 
    [author_id] => 1
    [featured] => 0
    [prim_key] => 2968
    [id_lang] => 2
    [title] => Squalenoyl siRNA PMP22 nanoparticles are effective in treating mouse models of C
    [paragraph] => Squalenoyl siRNA PMP22 nanoparticles are effective in treating mouse models of Charcot-Marie-Tooth disease type 1 A
    [content] => 
Authors
S Boutary, M Caillaud, M El Madani et al

 Lab
U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay, 94276, Le Kremlin-Bicêtre, France
Journal
Communications Biology
Abstract
Charcot-Marie-Tooth disease type 1 A (CMT1A) lacks an effective treatment. We provide a therapy for CMT1A, based on siRNA conjugated to squalene nanoparticles (siRNA PMP22-SQ NPs). Their administration resulted in normalization of Pmp22 protein levels, restored locomotor activity and electrophysiological parameters in two transgenic CMT1A mouse models with different severity of the disease. Pathological studies demonstrated the regeneration of myelinated axons and myelin compaction, one major step in restoring function of myelin sheaths. The normalization of sciatic nerve Krox20, Sox10 and neurofilament levels reflected the regeneration of both myelin and axons. Importantly, the positive effects of siRNA PMP22-SQ NPs lasted for three weeks, and their renewed administration resulted in full functional recovery. Beyond CMT1A, our findings can be considered as a potent therapeutic strategy for inherited peripheral neuropathies. They provide the proof of concept for a new precision medicine based on the normalization of disease gene expression by siRNA
BIOSEB Instruments Used
Grip strength test (BIO-GS3)
    [meta_description] => 
    [meta_keywords] => https://www.nature.com/articles/s42003-021-01839-2
    [meta_title] => 
    [link_rewrite] => squalenoyl-sirna-pmp22-nanoparticles-are-effective-in-treating-mouse-models-of-charcot-marie-tooth-disease-type-1-a
    [actif_langue] => 1
    [read] => 884
    [count_comments] => 0
    [id] => 1385
    [categories] => Array
        (
            [43] => Array
                (
                    [id_prestablog_categorie] => 43
                    [title] => Neurodégénérescences générales
                    [link_rewrite] => Neurodegenerescences-generales
                )

            [2] => Array
                (
                    [id_prestablog_categorie] => 2
                    [title] => Publications
                    [link_rewrite] => publications
                )

        )

    [authors] => 
    [paragraph_crop] => Squalenoyl siRNA PMP22 nanoparticles are effective in treating mouse models of [...]
    [link_for_unique] => 1
    [products_liaison] => Array
        (
            [48] => Array
                (
                    [name] => Test d'agrippement
                    [description_short] => 
Une méthode simple pour quantifier objectivement la force musculaire des rats et souris et l'effet de drogues, toxines, maladies musculaires (ex: myopathie) et neurodégénératives. Cette mesure de force est souvent employée en association avec le test de coordination motrice ROTAROD: un sujet présentant une coordination normale montrera des résultats médiocres en cas de faible force musculaire. Un must pour vos recherches sur l'activité, la coordination et le contrôle musculaire: particulièrement utile pour vos études sur les maladies de Parkinson et Huntington.
Nouveautés GS4 - 2023: Écran couleur rétroéclairé (meilleure lisibilité), pédale de remise à zéro, durée de batterie optimisée, taille de mémoire augmentée à 500 valeurs, compteur d'animaux, port USB (transfert de données/charge)

                    [thumb] => 
                    [img_empty] => /var/www/vhosts/de3310.ispfr.net/bioseb2024/modules/prestablog/views/img/product_link_white.jpg
                    [image_presente] => 1
                    [link] => https://bioseb.com/fr/activite-systeme-moteur-coordination/48-grip-strength-test.html
                )

        )

)
1