These CNVs may show
association with certain clinical conditions and help in predicting risk and also
diagnosis. All these approaches have their merits and demerits and in general are
tedious, time consuming and need manpower with stringent handling. This naturally
prompted the development of several automated methods for culturing cells in mass
scale, use of membrane bioreactors, and image analysis for interpreting the cytogenetic
In the last 10 years gene therapy has experienced a renascence thanks to the development of
safer and more efficient gene transfer vectors and to the advances in the cell therapy field.
This book brings together a comprehensive collection of gene therapy tools and their thera‐
peutic applications. The first part of the book covers different gene therapy vectors focusing
on their advantages and disadvantages.
Thyroid hormone receptors (TRs) play a major role in animal physiology.
TRs are important and very interesting regulators of diverse aspects, including
brain development, hearing, bone growth, morphogenesis, metabolism, intestine,
and heart rate in vertebrates (Fig. 1). Aberrant functions of TRs induce
tremendous defects in these pathways. For example, the human disease of
Resistance to Thyroid Hormone (RTH) (see Chapter 8 by Yoh and Privalsky)
is a genetically autosomal dominant inherited syndrome that is caused by mutations
in the gene encoding the TRβ.
Intracellularly expressed antibodies (intrabodies) have been used to inhibit
the function of various kinds of protein inside cells. However, problems
with stability and functional expression of intrabodies in the cytosol remain
unsolved. In this study, we show that single-chain variable fragment (scFv)
intrabodies constructed with a heavy chain variable (VH) leader signal
sequence at the N-terminus were translocated from the endoplasmic reti-culum into the cytosol of T lymphocytes and inhibited the function of
the target molecule, Wiskott–Aldrich syndrome protein (WASP)....
No one industry alone can provide the basis for major gains in renewable
resource chemical use. Although exciting research opportunities exist in areas
such as biopolymers, stereospecific molecules, new enzymes, novel materials,
and transgenic design, progress in isolated technical areas will not be sufficient.
We must take a broad view of future consumer needs and emphasize inter-
related research projects conducted in a parallel and coordinated manner.
The goals of experimental neuroscience research are fundamentally to gain mechanistic
understanding of the pathology of disease in order to identify appropriate
targets for potential pharmacological intervention and evaluation of putative therapies.
With the advances in gene manipulation and transgene technologies, we have
unprecedented ability to generate animal models of disease that more closely mimic
the clinical conditions.
Noninvasive techniques capable of investigating altered pathophysiologies are
now of paramount importance.