Cardiac function - page 2 Scientific Publications

Latest publication 09/21/2018

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
    [prim_key] => 2055
    [id_lang] => 1
    [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)
    [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] => 1363
    [count_comments] => 0
    [id] => 1047
    [categories] => Array
        (
            [100] => Array
                (
                    [id_prestablog_categorie] => 100
                    [title] => Cardiac function
                    [link_rewrite] => Cardiac-function
                )

            [53] => Array
                (
                    [id_prestablog_categorie] => 53
                    [title] => General motor functions
                    [link_rewrite] => General-motor-functions
                )

            [14] => Array
                (
                    [id_prestablog_categorie] => 14
                    [title] => Motor control
                    [link_rewrite] => Motor-control
                )

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

        )

    [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] => Grip strength test
                    [description_short] => 
An easy way to objectively quantify the muscular strength of mice and rats, and to assess the effect of drugs, toxins, muscular (i.e. myopathy) and neurodegenerative diseases on muscular degeneration. It is widely used in conjunction with the ROTAROD motor coordination test: a normally coordinated rodent will show a decreased latency to fall off the rotating rod if its muscular strength is low. The Grip Strength Test is a must for your research on activity, motor control & coordination, and is particularly well suited for studies on Parkinson's & Huntington's disease.
New features GS4 - 2023: Color display with permanent backlight screen for easier reading, reset by footswitch, Improved battery time, Larger data memory of 500 values, Animal counter, USB port (charging/data transfer)

                    [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/en/activity-motor-control-coordination/48-grip-strength-test.html
                )

        )

)
1