Draft Guidance for Industry Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products
... received a GT product for ornithine transcarbamylase deficiency (Ref. 4), late-onset T-cell leukemia in subjects who received a GT product for X-linked severe combined immunodeficiency (X-SCID) (Ref. 5), and development of tumors in the brain and spinal cord of a patient who received intrathecal all ...
... received a GT product for ornithine transcarbamylase deficiency (Ref. 4), late-onset T-cell leukemia in subjects who received a GT product for X-linked severe combined immunodeficiency (X-SCID) (Ref. 5), and development of tumors in the brain and spinal cord of a patient who received intrathecal all ...
It takes more than just a single target
... • Growing evidence indicates all HIV subtypes may be prone to errors; posing enormous challenges to viral load monitoring.2 • HIV-1 diversity is increasing and recombinants of greater complexity are being created.1,3 – Produces 1010 virions / day.4 – Creates a polymorphism every 2,000–5,000 nucl ...
... • Growing evidence indicates all HIV subtypes may be prone to errors; posing enormous challenges to viral load monitoring.2 • HIV-1 diversity is increasing and recombinants of greater complexity are being created.1,3 – Produces 1010 virions / day.4 – Creates a polymorphism every 2,000–5,000 nucl ...
Endocytobiosis and Cell Research
... nod gene inducers (Kape et al., 1992). Isoliquiritigenin exhibited an inducing activity already at 1 nM. This concentration is about 1 order of magnitude below those required of the previously described strong nod gene inducers daidzein and genistein. The stronger nod gene-inducing activity of isoli ...
... nod gene inducers (Kape et al., 1992). Isoliquiritigenin exhibited an inducing activity already at 1 nM. This concentration is about 1 order of magnitude below those required of the previously described strong nod gene inducers daidzein and genistein. The stronger nod gene-inducing activity of isoli ...
cece lesson - Canadian Healthcare Network
... cation) reabsorbed. Since there is no other pathway for effective chloride reabsorption in the duct, sodium is equally poorly reabsorbed and sweat emerging on the skin surface contains a high salt content.6 ...
... cation) reabsorbed. Since there is no other pathway for effective chloride reabsorption in the duct, sodium is equally poorly reabsorbed and sweat emerging on the skin surface contains a high salt content.6 ...
Mendelian inheritance - Center of Statistical Genetics
... people with normal stature are genotypically d/d, and the dwarf phenotype in principle could be D/d or D/D. However, it is believed that in D/D individuals the two "doses" of the D allele produce such a severe effect that this genotype is lethal. Therefore, all achondroplastics are heterozygotes. ...
... people with normal stature are genotypically d/d, and the dwarf phenotype in principle could be D/d or D/D. However, it is believed that in D/D individuals the two "doses" of the D allele produce such a severe effect that this genotype is lethal. Therefore, all achondroplastics are heterozygotes. ...
Complex Inheritance of the 5-Lipoxygenase Locus
... could be complicated by variations between the knockout and the control mice in the flanking region of 5LO, unrelated to the 5LO gene itself. That is, since the knockout was created on the strain 129 genetic background and then backcrossed to a B6 background by a series of crosses, the region around ...
... could be complicated by variations between the knockout and the control mice in the flanking region of 5LO, unrelated to the 5LO gene itself. That is, since the knockout was created on the strain 129 genetic background and then backcrossed to a B6 background by a series of crosses, the region around ...
Application to Modify an Approved Exempt or Notifiable Low Risk
... Please initial each of the following statements to indicate that you understand your responsibilities when dealing with GMOs and then sign the application form. I have read, considered and understand my responsibilities under the Gene Technology Act 2000 and agree to undertake the GMO dealing outlin ...
... Please initial each of the following statements to indicate that you understand your responsibilities when dealing with GMOs and then sign the application form. I have read, considered and understand my responsibilities under the Gene Technology Act 2000 and agree to undertake the GMO dealing outlin ...
A novel EVI1 gene family, MEL1, lacking a PR domain (MEL1S) is
... CBP-associated factor [P/CAF]),19 suggesting that EVI1 can function as a dual transcription regulator. On the other hand, it is reported that overexpression of EVI1 transforms rat fibroblast RAT1 cells,20 and it blocks transforming growth factor  (TGF-) signal transduction in mink lung cells21 and ...
... CBP-associated factor [P/CAF]),19 suggesting that EVI1 can function as a dual transcription regulator. On the other hand, it is reported that overexpression of EVI1 transforms rat fibroblast RAT1 cells,20 and it blocks transforming growth factor  (TGF-) signal transduction in mink lung cells21 and ...
Ingestion of bacterially expressed dsRNAs can produce specific and
... by a non-homologous spacer DNA. These con®gurations are summarized in Fig. 1 (top). 3.2. Improved effectiveness of bacteria-induced RNAi using modi®ed bacterial strains Escherichia coli strain BL21(DE3), used in the initial development of bacteria-induced RNAi (Timmons and Fire, 1998), was originall ...
... by a non-homologous spacer DNA. These con®gurations are summarized in Fig. 1 (top). 3.2. Improved effectiveness of bacteria-induced RNAi using modi®ed bacterial strains Escherichia coli strain BL21(DE3), used in the initial development of bacteria-induced RNAi (Timmons and Fire, 1998), was originall ...
... another study, 11 patients who were treated for an oropharyngeal squamous cell carcinoma were enrolled immediately after surgery in a pilot study to analyse effectiveness of a TheraBiteÒ apparatus.21 The mean increase in mouth opening after follow-up (12–48 weeks) was 9.7 mm. It is not clear, howeve ...
Leukaemia Section Chronic lymphocytic leukaemia (CLL) Atlas of Genetics and Cytogenetics
... uncommon disorders such as PLL, hairy-cell leukemia, mantle-cell lymphoma, and other lymphomas. Further, the Matutes score based on the most common marker profile in CLL, CD5+, CD23+, FMC7- and weak expression (+/-) of surface immunoglobulin (SIg) and CD22, can distinguish between typical and atypic ...
... uncommon disorders such as PLL, hairy-cell leukemia, mantle-cell lymphoma, and other lymphomas. Further, the Matutes score based on the most common marker profile in CLL, CD5+, CD23+, FMC7- and weak expression (+/-) of surface immunoglobulin (SIg) and CD22, can distinguish between typical and atypic ...
Analyzing the Paper
... only in women. Via quantitative trait locus mapping followed by a candidate gene strategy using both mutant mice and pharmacological tools, we now demonstrate that the melanocortin-1 receptor (Mc1r) gene mediates κ-opioid analgesia in female mice only. This finding suggested that individuals with va ...
... only in women. Via quantitative trait locus mapping followed by a candidate gene strategy using both mutant mice and pharmacological tools, we now demonstrate that the melanocortin-1 receptor (Mc1r) gene mediates κ-opioid analgesia in female mice only. This finding suggested that individuals with va ...
Chapter 14: MENDEL AND THE GENE IDEA
... The dominant allele is the one that is expressed (it exerts its phenotypic effect) in a heterozygote. It is identified with a uppercase (capital) letter, usually the first letter of the trait. Recessive Allele whose phenotypic effect is not observed in a heterozygote; its expression is masked by ...
... The dominant allele is the one that is expressed (it exerts its phenotypic effect) in a heterozygote. It is identified with a uppercase (capital) letter, usually the first letter of the trait. Recessive Allele whose phenotypic effect is not observed in a heterozygote; its expression is masked by ...
Protocadherin-1: epithelial barrier dysfunction in asthma and eczema Grissel Faura Tellez
... Asthma and eczema result from the interaction between genetic susceptibility and environmental exposures. Over the last two decades, genetic studies have led to the identification of multiple susceptibility genes, increasing our understanding of the biological pathways leading to these diseases. Man ...
... Asthma and eczema result from the interaction between genetic susceptibility and environmental exposures. Over the last two decades, genetic studies have led to the identification of multiple susceptibility genes, increasing our understanding of the biological pathways leading to these diseases. Man ...
Mutant Fruit Flies: Exploratorium Exhibit. Mutations in
... Click on the small thumbnail pictures below to magnify the flies. You'll see enlarged illustrations of fruit flies, Drosophila melanogaster. (In our real exhibit you'd be looking at the actual flies crawling around, looking for food or grooming their wings.) Compare the mutated flies to the normal f ...
... Click on the small thumbnail pictures below to magnify the flies. You'll see enlarged illustrations of fruit flies, Drosophila melanogaster. (In our real exhibit you'd be looking at the actual flies crawling around, looking for food or grooming their wings.) Compare the mutated flies to the normal f ...
Frequent, independent transfers of a catabolic gene from bacteria to
... We found as many as 65 acdS homologues in eukaryotes—four in stramenopiles (all oomycetes) and 61 in fungi—after analysis of 149 sequenced genomes based on the AcdS protein sequence of the proteobacterium Pseudomonas fluorescens F113. Significant sequence identity (at least 38% amino acid identity) ...
... We found as many as 65 acdS homologues in eukaryotes—four in stramenopiles (all oomycetes) and 61 in fungi—after analysis of 149 sequenced genomes based on the AcdS protein sequence of the proteobacterium Pseudomonas fluorescens F113. Significant sequence identity (at least 38% amino acid identity) ...
Daisy quorum drives for the genetic restoration of wild
... experimental plans for viewing and comment by the community before any experiments begin. As data are gathered and analyses completed, it will be updated with new figures, and eventually will become one or more peer-reviewed publications. All clinical trials now require pre-registration, and the “re ...
... experimental plans for viewing and comment by the community before any experiments begin. As data are gathered and analyses completed, it will be updated with new figures, and eventually will become one or more peer-reviewed publications. All clinical trials now require pre-registration, and the “re ...
2015 Explorer - UCLA School of Dentistry
... of narrow-diameter implants, and the higher mechanical strength of the TiZr alloy means that the risk for implant fractures associated with narrowdiameter implants could be minimized. While initial studies have shown that TiZr implants have excellent survival with minimal bone loss, further studies ...
... of narrow-diameter implants, and the higher mechanical strength of the TiZr alloy means that the risk for implant fractures associated with narrowdiameter implants could be minimized. While initial studies have shown that TiZr implants have excellent survival with minimal bone loss, further studies ...
Molecular ecology and selection in the drought-
... mechanistic complex trait. In terms of genetics, the multiple individual traits that make up drought tolerance are usually inherited quantitatively with very few major genes for drought tolerance mechanisms known, although Blair et al. [7] did find some quantitative trait loci for drought tolerance. ...
... mechanistic complex trait. In terms of genetics, the multiple individual traits that make up drought tolerance are usually inherited quantitatively with very few major genes for drought tolerance mechanisms known, although Blair et al. [7] did find some quantitative trait loci for drought tolerance. ...
The versatile worm: genetic and genomic resources for
... researchers has the potential to bewilder newcomers to the field. With this in mind, this Review provides a guide to the main resources that have been specifically developed for worm research. We first review online databases and tools, followed by a discussion of the most widely used experimental a ...
... researchers has the potential to bewilder newcomers to the field. With this in mind, this Review provides a guide to the main resources that have been specifically developed for worm research. We first review online databases and tools, followed by a discussion of the most widely used experimental a ...
“What is this Genetics, Anyway?” Understandings of
... clinical consultations, which identified potential disjunctures between clients’ and clinicians’ understandings. Emergent themes and issues, which could not have been anticipated at the outset, were then explored further in subsequent interviews. Participant observation enabled the first author to b ...
... clinical consultations, which identified potential disjunctures between clients’ and clinicians’ understandings. Emergent themes and issues, which could not have been anticipated at the outset, were then explored further in subsequent interviews. Participant observation enabled the first author to b ...
Understanding Dexamethasone and Other Steroids
... Adrenal glands produce hormones and steroids. The steroids influence many actions of the body’s systems. They are involved in regulation of carbohydrates, proteins, and fats. They also inhibit inflammatory, allergic, and normal immune responses. Synthetic versions can imitate the actions of the natu ...
... Adrenal glands produce hormones and steroids. The steroids influence many actions of the body’s systems. They are involved in regulation of carbohydrates, proteins, and fats. They also inhibit inflammatory, allergic, and normal immune responses. Synthetic versions can imitate the actions of the natu ...
Gene therapy
Gene therapy is the therapeutic delivery of nucleic acid polymers into a patient's cells as a drug to treat disease. Gene therapy could be a way to fix a genetic problem at its source. The polymers are either expressed as proteins, interfere with protein expression, or possibly correct genetic mutations.The most common form uses DNA that encodes a functional, therapeutic gene to replace a mutated gene. The polymer molecule is packaged within a ""vector"", which carries the molecule inside cells.Gene therapy was conceptualized in 1972, by authors who urged caution before commencing human gene therapy studies. By the late 1980s the technology had already been extensively used on animals, and the first genetic modification of a living human occurred on a trial basis in May 1989 , and the first gene therapy experiment approved by the US Food and Drug Administration (FDA) occurred on September 14, 1990, when Ashanti DeSilva was treated for ADA-SCID. By January 2014, some 2,000 clinical trials had been conducted or approved.Early clinical failures led to dismissals of gene therapy. Clinical successes since 2006 regained researchers' attention, although as of 2014, it was still largely an experimental technique. These include treatment of retinal disease Leber's congenital amaurosis, X-linked SCID, ADA-SCID, adrenoleukodystrophy, chronic lymphocytic leukemia (CLL), acute lymphocytic leukemia (ALL), multiple myeloma, haemophilia and Parkinson's disease. Between 2013 and April 2014, US companies invested over $600 million in the field.The first commercial gene therapy, Gendicine, was approved in China in 2003 for the treatment of certain cancers. In 2011 Neovasculgen was registered in Russia as the first-in-class gene-therapy drug for treatment of peripheral artery disease, including critical limb ischemia.In 2012 Glybera, a treatment for a rare inherited disorder, became the first treatment to be approved for clinical use in either Europe or the United States after its endorsement by the European Commission.