Efficient AAV9 Purification Using a Single-Step AAV9 Magnetic Affinity Beads Isolation.

Adeno-associated viruses (AAVs) have emerged as promising tools for gene therapy due to their safety and efficacy in delivering therapeutic genes or gene editing sequences to various tissues and organs. AAV serotype 9 (AAV9), among AAV serotypes, stands out for its ability to efficiently target multiple tissues, thus holding significant potential for clinical applications. However, existing methods for purifying AAVs are cumbersome, expensive, and often yield inconsistent results.

First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model.

The only treatment tested for growth hormone receptor (GHR) defective Laron Syndrome (LS) is injections of recombinant insulin-like-growth factor 1 (rhIGF1). The response is suboptimal and associated with progressive obesity. In this study, we treated 4-5-week-old Laron dwarf mice (GHR-/-) with an adeno-associated virus expressing murine GHR (AAV-GHR) injection at a dose of 4 × 10 vector genome per mouse.

Modification of a Constitutive to Glucose-Responsive Liver-Specific Promoter Resulted in Increased Efficacy of Adeno-Associated Virus Serotype 8-Insulin Gene Therapy of Diabetic Mice.

We have previously used a hepatotropic adeno-associated viral (AAV) vector with a modified human insulin gene to treat diabetic mice. The HLP (hybrid liver-specific promoter) used was constitutively active and non-responsive to glucose. In this study, we examined the effects of addition of glucose responsive elements (R3G) and incorporation of a 3' albumin enhancer (3'iALB) on insulin expression.

Correction of Murine Diabetic Hyperglycaemia With A Single Systemic Administration of An AAV2/8 Vector Containing A Novel Codon Optimized Human Insulin Gene.

We report the correction of hyperglycemia of STZ induced diabetic mice using one intravenous systemic administration of a single stranded serotype 8 pseudotyped adeno-associated virus (ssAAV2/8) vector encoding the human proinsulin gene under a constitutive liver specific promoter. In vivo dose titration experiments were carried out and we identified an optimal range that achieved maintenance of euglycaemia or a mild diabetic condition for at least 9 months and ongoing to beyond 1 year for some animals, accompanied by human C-peptide secretion and weight gain. Further DNA codon optimization of the insulin gene construct resulted in approximately 3-10 times more human C-peptide secreted in the blood of codon optimized treated animals thereby reducing the number of vector particles required to achieve the same extent of reduction in blood glucose levels as the non-codon optimized vector.

Effects of malnutrition on economic productivity in China as estimated by PROFILES.

Objective: To calculate the effects of malnutrition on economic productivity in China.

Methods: PROFILES was used to quantify the function consequences of malnutrition in term of protein energy malnutrition, iron deficiency and iodine deficiency.

Results: Productivity gained due to improved iodine nutrition.

Effects of malnutrition on child survival in China as estimated by PROFILES.

Objective: To estimate the benefits of reductions in underweight and Vitamin A deficiency for child survival in China that might be expected as a result of lowering the prevalence of these conditions.

Methods: Profiles, a process of nutrition policy analysis was used to quantify the functional consequences of malnutrition in terms of child survival.

Results: Underweight: The actual reduction in underweight between 1992 and 2001 (from 15.

[Epidemiological survey of chronic vascular complications of type 2 diabetic in-patients in four municipalities].

Objective: To provide an epidemiological description and risk factors of chronic vascular complications of type 2 diabetic in-patients in four municipalities including Beijing, Shanghai, Tianjin, and Chongqing.

Methods: Data of the study came from 3,469 Type 2 diabetic in-patients from 1991 to 2000 in 10 medical centers of Beijing, Shanghai, Tianjin, and Chongqing. A variety of parameters of in-patients were evaluated retrospectively to know the prevalence and risk factors of chronic vascular complications in the study patients.