Journal: Molecular Therapy. Methods & Clinical Development

Article Title: FAP-CAR-T cells reduce dystrophic muscle fibrosis, improving adeno-associated virus gene transfer efficacy

doi: 10.1016/j.omtm.2025.101545

Figure Lengend Snippet: Combined therapy: Gene transfer efficiency (A) Schematic representation of the in vivo protocol. Two-month-old D2.Mdx mice were injected or not with two consecutive doses (1 × 10 6 ) of control T cells or of FAP-CAR-T cells, and after 2 weeks of treatment the mice were injected with a single intravenous dose (5 × 10 12 VG/kg) of rAAV9-microdystrophin (μDystrophin or μDys). Tissues were collected 6 weeks after the AAV injection, 4 animals were included per group and statistical analyses used one-way ANOVA tests with p values indicated above the compared conditions. (B) Quantification of VGCN with ddPCR in TA, EDL, and DIA in DBA2-MDX mice. (C) Gene expression analysis of microdystrophin in TA, EDL, and DIA of DBA2-MDX mice. Results are shown as a histogram of relative abundance of gene expression over P0 normalizer gene. (D) Quantification of microdystrophin expression after capillary western blot with DYS-B antibody in TA, EDL, and DIA at 6 weeks post-injection in mice. Values are represented as normalized area under the curve (AUC). (E) Capillary western blot of protein lysates of TA at 6 weeks post-injection in mice. Microdystrophin are visible around 139 kDa. (F) Representative microdystrophin IHF pictures of the TA, EDL, and DIA at 6 weeks post-injection in mice. Images include a scale bar. (G) Quantification of dystrophin positive myofibers in TA, EDL, and DIA at 6 weeks post-injection in D2.mdx mice. The percentage of dystrophin+ fibers is represented of number of dystrophin positive fibers over fibers positive for laminin.

Article Snippet: The DBA2 (B6; 129S4-DBA2tm1Cpr/J, strain no. 000671) (D2) controls mice were purchased directly from Charles River Laboratories) while the DBA2/mdx (D2.B10-Dmdmdx/J, strain no. 013141) (D2.mdx) mice were bred and obtained from the Center d’Exploration et de Recherche Fonctionnelle Expérimentale (CERFE) in Evry, France.

Techniques: In Vivo, Injection, Control, Gene Expression, Expressing, Western Blot

Journal: Molecular Therapy. Methods & Clinical Development

Article Title: FAP-CAR-T cells reduce dystrophic muscle fibrosis, improving adeno-associated virus gene transfer efficacy

doi: 10.1016/j.omtm.2025.101545

Figure Lengend Snippet: Combined therapy: Histology effects Mice were treated as in A. (A) Representative images with scale bars, of hematoxylin and eosine, Sirius red, alizarine red coloration, and CD11b staining in the TA, EDL, and diaphragm of Dba2-mdx mice 6 weeks post-injection. For Sirius red coloration, red-colored regions are highly fibrotic and contain collagen deposits. For alizarine coloration, brown areas are indicated as a region containing calcium deposits. For CD11b staining, red signals indicate monocytes cells. (B) Quantification of the collagen, calcium deposits area and CD11b cells in the TA of mice. The results are shown as the percentage of fibrotic positive or calcium positive regions over the total area of the muscle cut for colorations. The results for CD11b staining are shown as the percentage of CD11b + cells after nuclei segmentation on R software. Statistical p values are indicated above the compared conditions.

Article Snippet: The DBA2 (B6; 129S4-DBA2tm1Cpr/J, strain no. 000671) (D2) controls mice were purchased directly from Charles River Laboratories) while the DBA2/mdx (D2.B10-Dmdmdx/J, strain no. 013141) (D2.mdx) mice were bred and obtained from the Center d’Exploration et de Recherche Fonctionnelle Expérimentale (CERFE) in Evry, France.

Techniques: Staining, Injection, Software

Journal: International journal of molecular sciences

Article Title: In Silico Structural Prediction for the Generation of Novel Performant Midi-Dystrophins Based on Intein-Mediated Dual AAV Approach.

doi: 10.3390/ijms251910444

Figure Lengend Snippet: Figure 4. Efficacy of reconstitution of midi-dystrophins in Dba2-mdx mouse model. (A) Schematic representation of the in vivo protocol. One month old Dba2/mdx mice were injected with midi-Dys 1, midi-Dys 2, and midi-Dys 3 at a dose of 2.0 × 1013 vg/kg each vector (total dose 4.0 × 1013 vg/kg).

Article Snippet: DBA2 (B6;129S4-DBA2tm1Cpr/J, strain #000671) and DBA2-mdx (D2.B10-Dmdmdx/J) mice were supplied by the Jackson Laboratory.

Techniques: In Vivo, Injection, Plasmid Preparation

Journal: International journal of molecular sciences

Article Title: In Silico Structural Prediction for the Generation of Novel Performant Midi-Dystrophins Based on Intein-Mediated Dual AAV Approach.

doi: 10.3390/ijms251910444

Figure Lengend Snippet: Figure 5. Functional and histopathological therapeutic efficacy in Dba2-mdx mice. (A) CPK (creatine phosphokinase) concentration in the sera of Dba2-mdx mice injected with midi-Dys 1-2-3. Results are

Article Snippet: DBA2 (B6;129S4-DBA2tm1Cpr/J, strain #000671) and DBA2-mdx (D2.B10-Dmdmdx/J) mice were supplied by the Jackson Laboratory.

Techniques: Functional Assay, Drug discovery, Concentration Assay, Injection