Coalition to Cure Calpain 3 is dedicated to empowering the scientific community as we advance effective treatments for calpainopathy. This toolbox provides researchers with essential resources to advance their work, facilitate collaboration and accelerate progress toward a cure.
Animal Models
| Model | Description |
| RNAless | CRISPR deletion of the capn3b promoter region + exon 1 (no expression) |
| mut1 | CRISPR deletion of capn3b exon 3-5, targeting the beginning of the calpain catalytic domain (no expression) |
| mut73 | CRISPR deletion of capn3b exon 3-5, targeting the beginning of the calpain catalytic domain (leaky phenotype) |
Contact: Berman Lab at the University of Ottawa, Sergey Prykhozhij (sprykhoz@uottawa.ca), or AGADA Biosciences, Pia Elustondo (pelustondo@agadabio.com)
| Model | Jax Strain | Availability | Notes |
| Capn3-deficient on S129 background | #031211 | Interested researchers can order cryo-recovery from Jax. C3 also maintains a colony of live mice. Contact us for more information. | Reference (PubMed) |
| Capn3-deficient on CC041 background | #031228 | Available by cryo-recovery | |
| Capn3-deficient on DBA/2J background | #031557 | Available by cryo-recovery | |
| Capn3-deficient on FVB/NJ background | #031227 | Available by cryo-recovery | |
| Capn3-deficient on NSG background | #038279 Donated by Vita Therapeutics | Available by cryo-recovery | Immunodeficient, permissive for xenograft/human tumor growth |
Antibodies
| Antibody | Host Species | Epitope | Species Reactivity | Notes |
| Leica CALP-12A2 | Mouse | Amino acids 355-370 in exon 8 of human sequence (Anderson et al) | Human, mouse, rabbit, dog, rat, chicken, hamster and pig | Recognizes CAPN3 (94 kDa) and ubiquitous CAPN1/2 (~60-70 kDa) in mouse, rat, chicken, and hamster lysates. |
| Leica CALP-2C4 | Mouse | Amino acids 1-19 in exon 1 of human sequence (Anderson et al) | Human, rabbit, dog, hamster. No reactivity to mouse, chicken, or pig. | N-terminal antibody that does not recognize 45-60 kDa CAPN3 fragments from autolysis (recognizes a 30 kDa band instead). |
| Proteintech 10415-1-AP | Rabbit | Amino acids 1-156 of human sequence | Human, mouse | |
| Proteintech 67366-1-Ig | Mouse | Amino acids 1-156 of human sequence | Human, mouse, rat, pig | |
| ThermoFisher PA5-103692 | Rabbit | Amino acids 257-307 of human sequence | Human, mouse, rat | Vendor claims this reacts with CAPN3. However, they use lung tissue lysate to demonstrate reactivity where there should be no CAPN3. |
Patient-Focused Drug Development
On September 23, 2022, a consortium of limb-girdle muscular dystrophy (LGMD) organizations held an Externally-Led Patient Focused Drug Development (EL-PFDD) meeting for six LGMD subtypes: 2A/R1, 2C/R5, 2D/R3, 2E/R4, 2F/R6, and 2I/R9. The consortium included the following organizations: Coalition to Cure Calpain 3, CureLGMD2I, Kurt+Peter Foundation, LGMD2D Foundation, McColl-Lockwood Laboratory for Muscular Dystrophy Research, and the Speak Foundation.
The EL-PFDD meeting was designed to engage patients and elicit their unique perspectives on the health effects, daily impacts, treatment goals, and decision factors considered when seeking out or selecting a treatment for symptoms and burdens associated with LGMD.
It is C3’s hope that drug developers and regulators will read the report and incorporate patients’ and caregivers’ perspectives when developing and reviewing new therapies to best meet the needs and expectations of our community.
LGMD2A/Calpainopathy Registry
The LGMD2A/Calpainopathy Registry collects disease-specific natural history data about individuals with Calpainopathy, with the goal of improving the understanding of Calpainopathy and informing treatment development.
As of July 2026, the Registry includes more than 400 global participants. Registry questionnaires were built from common data element standards and cover the following topics:
- Socio-demographics
- Medical history and diagnostics
- Treatment and disease progression
- Management of care
- Quality of life
- Clinical trial participation
We are interested in collaborating with you. If you would like access to the LGMD2A/Calpainopathy Registry findings for a research project, please contact our registry administrator at Registry@CureCalpain3.org for more information.
Access to the LGMD2A/Calpainopathy Registry data is contingent upon project approval by the LGMD2A/Calpainopathy Registry Advisory Board.