Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders.

Bioengineering of viral vectors for therapeutic gene delivery is a pivotal strategy to reduce doses, facilitate manufacturing, and improve efficacy and patient safety. Here, we engineered myotropic adeno-associated viral (AAV) vectors via a semirational, combinatorial approach that merges AAV capsid and peptide library screens. We first identified shuffled AAVs with increased specificity in the murine skeletal muscle, diaphragm, and heart, concurrent with liver detargeting.

A DNA telomerase vaccine for canine cancer immunotherapy.

Telomerase reverse transcriptase (TERT) is highly expressed in more than 90% of canine cancer cells and low to absent in normal cells. Given that immune tolerance to telomerase is easily broken both naturally and experimentally, telomerase is an attractive tumor associated antigen for cancer immunotherapy. Indeed, therapeutic trials using human telomerase peptides have been performed.

[Swallowing disorders and the elderly in a geriatric care facility].

It is essential to fight against malnutrition and dehydration in elderly people in care facilities. They result in the deterioration of the general status, a risk of a fall, a risk of wounds, an increased risk of infection and mortality in geriatrics. In addition, swallowing disorders due to ageing increase the risk of food going down the wrong way.

Anticancer DNA vaccine based on human telomerase reverse transcriptase generates a strong and specific T cell immune response.

Human telomerase reverse transcriptase (hTERT) is overexpressed in more than 85% of human cancers regardless of their cellular origin. As immunological tolerance to hTERT can be overcome not only spontaneously but also by vaccination, it represents a relevant universal tumor associated antigen (TAA). Indeed, hTERT specific cytotoxic T lymphocyte (CTL) precursors are present within the peripheral T-cell repertoire.

[Waardenburg's syndrome and severe cyanotic cardiopathy].

Waardenburg syndrome type I is transmitted as an autosomal dominant trait. The main features are dystopia canthorum, partial pigmentary disorder and perceptive deafness. Osteo-articular and intestinal malformations may be observed.