AAV2/PHP.eB-CBh-eGFP-WPRE-SV40pA = 6.9e12 vg/mL
AAV2/PHP.eB-hSYN-eGFP-WPRE-hGH = 1.2e13 vg/mL
AAV2/PHP.eB-hSYN-C8S9-HA-FokI NEL-eGFP-9.5RVD = 5.2e12 vg/mL
AAV2/PHP.eB-hSYN-C8S9-Flag-FokI CKK-11.5RVD = 3.9e13 vg/mL
Toe DNA of heteroplasmic tRNAAla mice were obtained at P5, before performing the injections to determine basal levels of heteroplasmy. For systemic delivery, mice at P16/18 were subjected to retro-orbital injection with 2.0–4.0e13 vg/kg in a 40-μL final volume in saline. When injecting mitoTALEN, both monomers were delivered together (total 4e13 (link) vg/kg). All injections were carried out using a short insulin syringe with a 31G needle (Becton Dickenson). Tail tissue was obtained before injection. At 6, 12, and 24 weeks after injection, mice were deeply anesthetized and perfused with chilled PBS, and skeletal, cardiac tissues, liver, kidney samples and different areas of the brain (cortex, cerebellum, hippocampus, striatum/midbrain, and spinal cord) were obtained.