Comprehensive Risk Assessment of LAD Disease Progression in CCTA: The CLAP Score Study.

Background: a wider left main bifurcation angle (LMBA) has been linked to severe plaque development in the proximal left anterior descending artery (LAD). This study aimed to identify predictors of severe proximal LAD stenosis and major adverse cardiovascular events (MACE) using coronary computed tomography angiography (CCTA).

Methods: from an initial cohort of 650 consecutive patients, we analyzed 499 patients who met the inclusion criteria after exclusions.

Combined intraocular and intravenous gene delivery for therapy of gyrate atrophy of the choroid and retina.

Gyrate atrophy of the choroid and retina (GACR) is due to ornithine aminotransferase (OAT) deficiency, which causes hyperornithinemia, leading to retinal pigment epithelium, followed by choroidal and retinal degeneration. Adeno-associated virus serotype 8 (AAV8) vector-mediated OAT (AAV8-OAT) liver gene transfer reduces ornithinemia in the Oat mouse model of GACR and improves retinal function and structure. Since OAT is expressed in various tissues including the retina, we investigated the efficacy of restoration of OAT expression in either retina or liver or both tissues on the retinal phenotype of Oat mice.

Transcrestal maxillary sinus floor elevation with injectable xenogeneic bone substitute in gel form: A clinical, radiological and histological analysis.

Purpose: This retrospective study measured the increase in bone tissue using the transcrestal maxillary sinus floor elevation with injectable xenogeneic bone substitute in gel form with simultaneous implant placement. This procedure allows elevation of the sinus floor atraumatically, reducing the risk of perforation of the Schneiderian membrane.

Methods: 52 subjects needing unilateral sinus floor elevation, with a residual crestal height from 2 mm to 5 mm, and a request for at least one implant-prosthetic rehabilitation in the posterior maxillary area were enrolled.

Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases.

Liver-directed adeno-associated viral (AAV) vector-mediated homology-independent targeted integration (AAV-HITI) by CRISPR-Cas9 at the highly transcribed albumin locus is under investigation to provide sustained transgene expression following neonatal treatment. We show that targeting the 3' end of the albumin locus results in productive integration in about 15% of mouse hepatocytes achieving therapeutic levels of systemic proteins in two mouse models of inherited diseases. We demonstrate that full-length HITI donor DNA is preferentially integrated upon nuclease cleavage and that, despite partial AAV genome integrations in the target locus, no gross chromosomal rearrangements or insertions/deletions at off-target sites are found.

Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B.

Gene therapy of Usher syndrome type 1B (USH1B) due to mutations in the large () gene is limited by the packaging capacity of adeno-associated viral (AAV) vectors. To overcome this, we have previously developed dual AAV8 vectors which encode human (dual AAV8.).