This introductory chapter provides a short review of the ideas and practical approaches that have led to the present and perceived future development of prenatal gene therapy. It summarizes the advantages and the potential adverse effects of this novel preventive and therapeutic appr ...

This chapter gives a comparative review of the different vector systems applied to date in prenatal gene therapy experiments highlighting the need for versatility and choice for application in accordance with the actual aim of the study. It reviews the key characteristics of the four main ge ...

Vectors based on adeno-associated virus (AAV) show great promise for safe, efficacious therapeutic gene transfer in extensive pre-clinical data and, recently, in clinical trials. Careful vector design and choice from a range of natural or synthetic pseudotypes allow targeted, effic ...

Adenoviruses have many attributes, which have made them one of the most widely investigated vectors for gene therapy applications. These include ease of genetic manipulation to produce replication-deficient vectors, ability to readily generate high titer stocks, efficiency of g ...

Vectors derived from the Retroviridae family have several attributes required for successful gene delivery. Retroviral vectors have an adequate payload size for the coding regions of most genes; they are safe to handle and simple to produce. These vectors can be manipulated to target diff ...

Gene therapy vectors based on viruses are the most effective gene delivery systems in use today and although efficient at gene transfer their potential toxicity (Hacein-Bey-Abina et al., Science 302:415–419, 2003) provides impetus for the development of safer non-viral alternatives. An ...

Prenatal stem cell therapy has broad potential for therapeutic application. “Stem cells” of interest include multipotent adult-derived stem cells, cord blood, amniotic fluid, or fetal stem cells, and embryonic or induced pluripotent stem cells. Potential manipulations of stem cel ...

Testing in animal models is an essential requirement during development of prenatal gene therapy for �clinical application. Some information can be derived from cell lines or cultured fetal cells, such as the efficiency of gene transfer and the vector dose that might be required. Fetal tiss ...

Quantitative trait locus (QTL) analysis is a statistical method to link phenotypes with regions of the genome that affect the phenotypes in a mapping population. R/qtl is a powerful statistical program commonly used for analyzing rodent QTL crosses, but R/qtl is a command line program that can be ...

Different expression of maternally and paternally inherited alleles at certain genes is called genetic imprinting. Despite its great importance in trait formation, development, and evolution, it remains unclear how genetic imprinting operates in a complex network of interacti ...

Haplotype Association Mapping (HAM) is a novel phenotype-driven approach to identify genetic loci and was originally developed for mice. This method, which is similar to Genome-Wide Association (GWA) studies in humans, looks for associations between the phenotype and the haplotypes ...

Candidate gene association study is the most common method for associating human genetic variations with the phenotypes they produce, due to the relative simplicity of acquiring patient samples and genotype data. The study design begins with identifying appropriate DNA samples and an ...

Genome-wide association studies have opened a new era in the study of the genetic basis of common, multifactorial diseases and traits. Before the introduction of this approach only a handful of common genetic variants showed consistent association for any phenotype. Using genome-wide a ...

Since the introduction of genetical genomics in 2001, many studies have been published on various organisms, including mouse and rat. Genetical genomics makes use of the latest microarray profiling technologies and combines vast amounts of genotype and gene expression information, a ...

Gene expression profiling provides unprecedented opportunities to study patterns of gene expression regulation, for example, in diseases or developmental processes. Bioinformatics analysis plays an important part of processing the information embedded in large-scale e ...

Improving human health is a major aim of medical research, but it requires that variation between individuals be taken into account since each person carries a different combination of gene variants and is exposed to different environmental conditions, which can cause differences in sus ...

It is increasingly common to combine genome-wide expression data with quantitative trait mapping data to aid in the search for sequence polymorphisms responsible for phenotypic variation. By joining these complex but different data types at the level of the biological pathway, we can ta ...

Mouse models have become the most common model for defining mechanisms of atherosclerotic disease. Many genetic manipulations have enabled the development of atherosclerosis in mice due to either endogenous or diet-induced hypercholesterolemia. This availability of lesion ...

Development of effective treatment or, more critically, preventative measures against atherosclerosis and cardiovascular disease will require animal models that mimic the disease processes seen in humans and permit identification of the genetic and physiological factor ...

Hypertension is a leading cause of heart attack, stroke, and kidney failure and represents a serious medical issue worldwide. The genetic basis of hypertension is well-established, but few causal genes have been identified thus far. Non-invasive blood pressure measurements are a criti ...