Abstract：
:The eye is an excellent target organ for gene therapy. It is physically isolated, easily accessible, immune-privileged, and postmitotic. Furthermore, potential gene therapies introduced into the eye can be evaluated by noninvasive methods such as fundoscopy, electroretinography, and optical coherence tomography. In the last two decades, great advances have been made in understanding the molecular underpinnings of retinal degenerative diseases. Building upon the development of modern techniques for gene delivery, many gene-based therapies have been effectively used to treat loss-of-function retinal diseases in mice and men. Significant effort has been invested into making gene delivery vehicles more efficient, less toxic, and non-immunogenic. However, one challenge for the treatment of more complex gain-of-function diseases, many of which might be benefited by the regulation of cellular stress-responsive signaling pathways, is the ability to control the strategy in a physiological (conditional) manner. This review is focused on promising retinal gene therapy strategies that rely on small molecule-based conditional regulation and the inherent limitations and challenges of these strategies that need to be addressed prior to their extensive use.
journal_name
Adv Exp Med Bioljournal_title
Advances in experimental medicine and biologyauthors
Datta S,Peng H,Hulleman JDdoi
10.1007/978-3-030-27378-1_11subject
Has Abstractpub_date
2019-01-01 00:00:00pages
65-69eissn
0065-2598issn
2214-8019journal_volume
1185pub_type
杂志文章,评审abstract：:Recent studies emphasize the role of chronic hypoxia in the kidney as a final common pathway to end-stage renal disease (ESRD). Hypoxia of tubular cells leads to apoptosis or epithelial-mesenchymal transdifferentiation. This in turn exacerbates fibrosis of the kidney with loss of peritubular capillaries and subsequent...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-0-387-75434-5_7
更新日期：2007-01-01 00:00:00
abstract：:The silicone tube model of regeneration has proved to be an invaluable tool for experimental studies aimed at understanding expression of growth factors during normal and abnormal metabolic states of regeneration. Since the morphological parameters of nerve growth and myelination are well-defined and easily identified...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-1-4615-3448-8_5
更新日期：1992-01-01 00:00:00
abstract：:The gene coding for the surface spike protein gp195 of TGEV has been cloned and expressed in E. coli in the form of fusion proteins. These proteins were isolated and used to immunize laboratory animals. All animals developed antibodies cross-reacting with the TGEV virion but failed to neutralize the virus. The entire ...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-1-4684-7974-4_4
更新日期：1985-01-01 00:00:00
abstract：:Studies on the interaction of the green tea polyphenol (-)-Epigallocatechin-3-gallate (EGCG) with fourteen disease-related amyloid polypeptides and prions Huntingtin, Amyloid-beta, alpha-Synuclein, islet amyloid polypeptide (IAPP), Sup35, NM25 and NM4, tau, MSP2, semen-derived enhancer of virus infection (SEVI), immun...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-3-319-18365-7_7
更新日期：2015-01-01 00:00:00
abstract：:The expression of functional proteins in heterologous hosts is a core technique of modern biotechnology. The transfer to a suitable expression system is not always achieved easily because of several reasons: genes from different origins might contain codons that are rarely used in the desired host or even bear noncano...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-0-387-75532-8_5
更新日期：2007-01-01 00:00:00
abstract：:Suppressor monocytes have been found in a number of human diseases most of which are associated with lymphopenia and deficiences in cell mediated immunity. In our studies both quantitative and qualitative differences in monocytes were detected in certain patients with advanced Hodgkin's disease or tuberculosis. In cer...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-1-4684-8914-9_27
更新日期：1979-01-01 00:00:00
abstract：:In an attempt to identify the binding sites of myosin fragments to the nitrocellulose film in a flow cell, proteolytic digestion was performed of myosin fragments bound to the substrate. Chymotryptic digestion of papain.Mg-subfragment-1 (S1) showed that a large proportion of S1 were bound near the tail and that the di...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-1-4615-2872-2_25
更新日期：1993-01-01 00:00:00
abstract：:High-field (500 MHz) proton NMR has been used to elucidate the primary and secondary structures of glycosphingolipids (GSLs). Using 2-D J-correlated spectroscopy (2-D SECSY) which establishes scalar couplings of protons, the monosaccharide composition, anomeric configuration and aglycon structures of a GSL can be esta...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-1-4684-1200-0_8
更新日期：1984-01-01 00:00:00
abstract：:Dynamic changes in gene expression punctuate lymphocyte development and are a characteristic of lymphocyte activation. A prevailing view has been that these changes are driven by DNA transcription factors, which are the dominant force in gene expression. Accumulating evidence is challenging this DNA centric view and h...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-1-4419-5632-3_1
更新日期：2011-01-01 00:00:00
abstract：:RNA virus quasispecies are subjected to processes of positive Darwinian selection, to a very active and continuous negative selection and to random genetic drift. The course of RNA virus evolution is often unpredictable, and recent results suggest that even highly conserved motifs, once regarded as essential for infec...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-1-4615-5331-1_93
更新日期：1998-01-01 00:00:00
abstract：:The use of targeted proteomics to identify urinary biomarkers of kidney disease in urine can avoid the interference of serum proteins. It may provide better sample throughput, higher sensitivity, and specificity. Knowing which urinary proteins to target is essential for targeted proteomics. In perfusates, there were p...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-94-017-9523-4_17
更新日期：2015-01-01 00:00:00
abstract：:The extraordinary mechanical properties of spider silk fibers result from the interplay of composition, structure and self-assembly of spider silk proteins (spidroins). Genetic approaches enabled the biotechnological production of recombinant spidroins which have been employed to unravel the self-assembly and spinning...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-981-13-9791-2_6
更新日期：2019-01-01 00:00:00
abstract：:Studies in this laboratory on the nuclear binding sites (acceptors) for progesterone receptor in the developed chick oviduct have resulted in the detection of seasonal variations in the levels and functions of the receptor. Cytosol preparations obtained from the chick oviducts during the winter/spring period between J...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-1-4757-6589-2_13
更新日期：1979-01-01 00:00:00
abstract：UNLABELLED:Polychlorinated biphenyls (PCBs) and dioxins are environmental pollutants. Prenatally, as well as postnatally through breast feeding, large amounts are transferred from mother to the child. Formula is free of these substances. Considering their potential developmental neurotoxicity, we investigated long term...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：
更新日期：2000-01-01 00:00:00
abstract：:In summary, we have demonstrated that the ocular surface epithelia express at least three mucin genes. We suggest that the gel-forming mucin MUC5AC is a major mucin forming the mucus gel of the tear film. We further suggest that MUC1 facilitates the spread of the MUC5-containing mucus on the ocular surface and, along ...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-1-4615-5359-5_32
更新日期：1998-01-01 00:00:00
abstract：:Effects of DMSO on heme synthesis and enzymes of the heme biosynthetic pathway were examined in human HepG2 hepatoma cells. HepG2 cells contain measurable levels of ALA synthase and ALA dehydratase, and their levels are increased after treatment of cells with DMSO. DMSO treatment also led to increases in heme content ...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-1-4613-0623-8_12
更新日期：1989-01-01 00:00:00
abstract：:Antibacterial photodynamic therapy (APDT) is a promising method of treating local infected foci, in particular, surgical and burn wounds, trophic and diabetic ulcers. Photodynamic inactivation (PDI) is able to effectively destroy bacterial cells without them developing resistance in response to treatment.This work was...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/5584_2019_431
更新日期：2020-01-01 00:00:00
abstract：:Loudness perception can be studied based on the assumption that sounds of equal loudness elicit equal reaction time (RT; or "response latency"). We measured the underwater RTs of a harbor porpoise to narrowband frequency-modulated sounds and constructed six equal-latency contours. The contours paralleled the audiogram...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-1-4939-2981-8_153
更新日期：2016-01-01 00:00:00
abstract：:Cruciferous plant foods contain large quantities of secondary plant metabolites that have been shown to inhibit chemically induced carcinogenesis in animals. One mechanism by which these chemicals may inhibit carcinogenesis is through the induction of enzymes, such as cytochrome P-450-dependent monooxygenases, glutath...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-1-4899-2626-5_13
更新日期：1991-01-01 00:00:00
abstract：:Tumors consists of subpopulation of cells in which each subtype has contributes to tumor progression. Specifically one subtype known as cancer stem cells are associated with the initiation, progression, resistance to conventional therapies and metastasis. Metastasis is leading cause of cancer related deaths. Overall i...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/5584_2018_279
更新日期：2018-01-01 00:00:00
abstract：:1) The immunological mechanism of protection against dental caries has been investigated by passive immunization with antisera to S. mutans in the rhesus monkey. 2) Passive immunization with separated IgG, IgM and IgA from sera of immunized monkeys supports the concept that IgG, unlike IgA or IgM antibodies are protec...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-1-4684-3369-2_35
更新日期：1978-01-01 00:00:00
abstract：:Before a messenger RNA (mRNA) is translated into a protein in the cytoplasm, its pre-mRNA precursor is extensively processed through capping, splicing and polyadenylation in the nucleus. Defects in the processing of pre-mRNAs due to mutations in RNA sequences often cause disease. Traditional small molecules or protein...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-1-4939-1221-6_9
更新日期：2014-01-01 00:00:00
abstract：:Upregulation of angiogenesis is a frequent occurrence in lung cancer and is reported to represent a negative prognostic factor. This provides a rationale for the development and evaluation of anti-angiogenic agents. To date bevacizumab, a monoclonal antibody directed against serum VEGF, is the only anti-angiogenic age...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-3-319-24223-1_5
更新日期：2016-01-01 00:00:00
abstract：:Sex differences in cardiovascular diseases can be classified as those which are specific to one sex and those that differ in incidence, prevalence, etiology, symptomatology, response to treatment, morbidity, and mortality in one sex compared to the other. All sex differences in cardiovascular conditions have their bas...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-3-319-77932-4_27
更新日期：2018-01-01 00:00:00
abstract：:Functional oxygen-17 magnetic resonance (MR) imaging and localized spectroscopy is defined as the ensemble of MR measurements aiming at in vivo, noninvasive characterization of oxygen transport and utilization. After a brief description of the present status of in vivo 17O-MR, preliminary results are reported on oxyge...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-1-4615-0205-0_35
更新日期：2003-01-01 00:00:00
abstract：:Arrays of electronic sensors, capable of detecting and differentiating complex mixtures of volatile compounds, have been utilized to differentiate aromas of food and related materials. These sensor arrays have been dubbed "Electronic Noses" and have been commercially available in the USA for the past 4-5 years. Electr...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-1-4615-1247-9_5
更新日期：2001-01-01 00:00:00
abstract：:The clinical challenge on surface engineering of medical devices to prevent microorganisms adhesion and biofilm formation, has become an essential aspect for medical implants. Antibacterial properties of Graphene Oxide (GO) have been demonstrated across a broad spectrum of bacteria, and the different mechanisms of act...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/5584_2019_434
更新日期：2020-01-01 00:00:00
abstract：:The use ofneonicotinoid insecticides has grown considerably since their introduction in 1990s. They are used extensively for the control of agriculturally important crop pests and also in the control of cat and dog fleas. Imidacloprid exploited through an elaborated structural and substituent optimization of nithiazin...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：
更新日期：2010-01-01 00:00:00
abstract：:Assessment is a key element in education. Given its multiple methods, assessment contains the aspect of correctness and honesty from both the assessor and the assessed. These methods can be classified and their types are numerous, starting from single questions to tests and practical work. Concerning tests, their vast...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章
doi：10.1007/978-3-319-56246-9_6
更新日期：2017-01-01 00:00:00
abstract：:In this chapter, we intend to review gene therapy concepts applied to the potential treatment of tyrosinemia for parents and pediatricians. Therefore, our main objective is to give general informations in a comprehensible manner. Considering the nature of tyrosinemia and the current state of technology, a particular f...
journal_title：Advances in experimental medicine and biology
pub_type： 杂志文章,评审
doi：10.1007/978-3-319-55780-9_21
更新日期：2017-01-01 00:00:00
