Fonction cardiaque - page 2 - Publications Scientifiques

Dernière publication 21/09/2018

A Direct Comparison of IV and ICV Delivery Methods for Gene Replacement Therapy

Spinal muscular atrophy with respiratory distress type 1 (SMARD1) is an infantile autosomal recessive disease caused by the loss of the...

Array
(
    [id_prestablog_news] => 1047
    [id_shop] => 1
    [date] => 2018-09-21 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
    [ishero] => 0
    [prim_key] => 2056
    [id_lang] => 2
    [title] => A Direct Comparison of IV and ICV Delivery Methods for Gene Replacement Therapy 
    [paragraph] => A Direct Comparison of IV and ICV Delivery Methods for Gene Replacement Therapy in a Mouse Model of SMARD1.
    [content] => 
Authors
Shababi M, Villal—n E, Kaifer KA, DeMarco V, Lorson CL

 Lab
Department of Veterinary Pathobiology, College of Veterinary Medicine, University of Missouri, Columbia, MO, USA.
Journal
Molecular Therapy Methods  and Clinical Development
Abstract
Spinal muscular atrophy with respiratory distress type 1 (SMARD1) is an infantile autosomal recessive disease caused by the loss of the ubiquitously expressed IGHMBP2 gene. SMARD1 causes degeneration of alpha-motor neurons, resulting in distal muscle weakness, diaphragm paralysis, and respiratory malfunction. We have reported that delivery of a low dose of AAV9-IGHMBP2 to the CNS results in a significant rescue of the SMARD1 mouse model (nmd). To examine how a delivery route can impact efficacy, a direct comparison of intravenous (IV) and intracerebroventricular (ICV) delivery of AAV9-IGHMBP2 was performed. Using a low-dose, both IV and ICV delivery routes led to a significant extension in survival and increased body weight. Conversely, only ICV-treated animals demonstrated improvements in the hindlimb muscle, neuromuscular junction, and motor function. The hindlimb phenotype of IV-treated mice resembled the untreated nmd mice. We investigated whether the increased survival of IV-treated nmd mice was the result of a positive impact on the cardiac function. Our results revealed that cardiac function and pathology were similarly improved in IV- and ICV-treated mice. We concluded that while IV delivery of a low dose does not improve the hindlimb phenotype and motor function, partial restoration of cardiac performance is sufficient to significantly extend survival.
BIOSEB Instruments Used
Grip strength test (BIO-GS3)

Keywords/Topics
Functions de motricité générale; Système moteur; Fonction cardiaque
    [meta_description] => 
    [meta_keywords] => https://www.cell.com/molecular-therapy-family/methods/pdf/S2329-0501(18)30082-2.pdf
    [meta_title] => 
    [link_rewrite] => a-direct-comparison-of-iv-and-icv-delivery-methods-for-gene-replacement-therapy-in-a-mouse-model-of-smard1-
    [actif_langue] => 1
    [read] => 1442
    [count_comments] => 0
    [id] => 1047
    [categories] => Array
        (
            [100] => Array
                (
                    [id_prestablog_categorie] => 100
                    [title] => Fonction cardiaque
                    [link_rewrite] => Fonction-cardiaque
                )

            [53] => Array
                (
                    [id_prestablog_categorie] => 53
                    [title] => Functions de motricité générale
                    [link_rewrite] => Functions-de-motricite-generale
                )

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

            [14] => Array
                (
                    [id_prestablog_categorie] => 14
                    [title] => Système moteur
                    [link_rewrite] => Systeme-moteur
                )

        )

    [authors] => 
    [paragraph_crop] => A Direct Comparison of IV and ICV Delivery Methods for Gene Replacement Therapy in a Mouse [...]
    [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