" Development of Primary Cultures of Diaphragmatic Myoblasts for Basic Research Purposes "
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: During the surgery planned as part of the routine care of the patient, a part of the tissue sample will be saved in order to carry out the analysis planned for the research..
- Who it may be relevant to
- Registry conditions: Endometriosis. Basic parameters: from 18 years · Female.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- France
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
Neuromuscular diseases (NMDs) affecting motor neurons (MN) induce progressive muscle denervation, and become fatal when respiratory muscles e.g. the diaphragm are affected and can no longer contract. In some cases, such as Charcot's disease (Amyotrophic lateral sclerosis-ALS), there is no cure and patients die due to respiratory failure few years after diagnosis. Investigations on NMD-induced alterations of respiratory muscles in humans are limited notably by the absence of available in vitro model based on cell cultures of diaphragm-derived myoblasts. Yet, this cell tool is likely to help in developing original therapies to limit diaphragm muscle atrophy and dysfunction in NMD. To date, only cell cultures of human myoblasts obtained from limb muscles are available, making difficult to transpose results to the diaphragm. Thus, in the present project, we propose to : 1. originally develop primary cultures of myoblasts from human diaphragm, obtained from surgical resection of diaphragmatic endometriosis, 2. characterize them in terms of differentiation status (Histology, IF), metabolism (Metabolomics by NMR, cell respiration), and gene expression (RNASeq), in comparison to primary myoblasts cultures derived from the deltoid already available in the team. This project will provide an original new tool and important data on the specificity of diaphragm-derived myoblasts, compared to limb muscle-derived myoblasts with the long-term perspective of opening new therapeutical pathways for patients with severe NMDs.
Detailed description
Neuromuscular diseases (NMDs) affecting motor neurons (MN) induce progressive muscle denervation, and become fatal when respiratory muscles e.g. the diaphragm are affected and can no longer contract. In some cases, such as Charcot's disease (Amyotrophic lateral sclerosis-ALS), there is no cure and patients die due to respiratory failure few years after diagnosis. Investigations on NMD-induced alterations of respiratory muscles in humans are limited notably by the absence of available in vitro model based on cell cultures of diaphragm-derived myoblasts. Yet, this cell tool is likely to help in developing original therapies to limit diaphragm muscle atrophy and dysfunction in NMD. To date, only cell cultures of human myoblasts obtained from limb muscles are available, making difficult to transpose results to the diaphragm.
Thus, in the present project, we propose to :
1. originally develop primary cultures of myoblasts from human diaphragm, obtained from surgical resection of diaphragmatic endometriosis, 2. characterize them in terms of differentiation status (Histology, IF), metabolism (Metabolomics by NMR, cell respiration), and gene expression (RNASeq), in comparison to primary myoblasts cultures derived from the deltoid already available in the team.
This project will provide an original new tool and important data on the specificity of diaphragm-derived myoblasts, compared to limb muscle-derived myoblasts with the long-term perspective of opening new therapeutical pathways for patients with severe NMDs.
Interventions
- Other During the surgery planned as part of the routine care of the patient, a part of the tissue sample will be saved in order to carry out the analysis planned for the research.
During the surgery planned as part of the routine care of the patient, a part of the tissue sample will be saved in order to carry out the analysis planned for the research.
Primary outcome measures
- Molecular characteristics [Time frame: Inclusion]
- Cellular characteristics [Time frame: Inclusion]
Secondary outcome measures (1)
- Presence of diaphragm-derived myoblasts [Time frame: Inclusion]
Eligibility criteria
Inclusion criteria
- Age ≥ 18 years
- diaphragmatic endometriosis requiring surgical resection of the nodules in the diaphragm.
- Collection of non-opposition
Exclusion criteria
- Inability to speak and/or read French
- Patients under tutor or curatorship
- Protected adults
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
France · 1 center
- Department of Thoracic Surgery - Cochin Hospital — Paris
Identifiers
NCT: NCT07380308 · APHP250977 · 2024-A02556-41