• When a scientist changes a gene it is called recombinant technology. (thetech.org)
  • This gene encodes a nuclear polyglutamine-binding protein that is involved with transcription activation. (nih.gov)
  • Multiple alternatively spliced transcript variants that encode different protein isoforms have been described for this gene. (nih.gov)
  • Gene expression was analyzed by measuring beta-galactosidase activity in tissue homogenates or visualizing enzymatic activity or protein production at a cellular level by in situ histochemistry or immunocytochemistry. (nih.gov)
  • in an operon with an upstream PurR/LacI-type transcriptional regulator gene, named amlR ( ACSP50_2475 ), and a gene downstream ( ACSP50_2473 ) encoding a GGDEF-EAL-domain-containing protein putatively involved in c-di-GMP signaling. (frontiersin.org)
  • In order to investigate the functional role of miR-210 during TDI sensitization, BALBc mice were dermally exposed to TDI (0.5-4% v/v) and gene and protein expression were evaluated in the draining lymph nodes (dLN) and ears using RT-PCR and Western blot/ flow cytometry, respectively. (cdc.gov)
  • A gene is a piece of DNA that has instructions for making a protein. (thetech.org)
  • The enzyme consists of a protein component with reverse transcriptase activity, encoded by this gene, and an RNA component which serves as a template for the telomere repeat. (thermofisher.com)
  • Findings from his research include the transporter molecule responsible for P. falciparum chloroquine resistance (PfCRT), the molecules responsible for antigenic variation and immune evasion by P. falciparum (var genes), a mechanism for sickle-cell trait protection against malaria, and the PfHRP-II protein used for malaria rapid diagnostic tests (RDTs). (nih.gov)
  • In the present study, we evaluated the efficiency of protein translation, focusing on the effect of DNA templates of different lengths on cell-free protein synthesis using a luciferase assay. (nature.com)
  • The expression level from luc 25.7k is 1000-times higher than that with luc 1.7k, indicating that the protein yield per target gene is increased by 1000-times of magnitude for a longer template, luc 25.7k (Fig. 1 d). (nature.com)
  • FUJIFILM Diosynth Biotechnologies assists in the manufacturing of recombinant protein vaccines used for multiple indications including Covid-19. (fujifilm.com)
  • In order to identify key regulators of taxol pathway, cDNAs will be explore to obtain the recombinant TF protein. (europa.eu)
  • LIM domain only 1 (LMO1) is a protein-coding gene. (prospecbio.com)
  • hence the protein may regulate transcription by competitively binding to specific DNA-binding transcription factors. (prospecbio.com)
  • Before genomics, the path to a biotech drug typically began when scientists identified the gene that coded for an important, well-studied protein, such as insulin. (technologyreview.com)
  • With the genetic recipe in hand, they could use recombinant DNA techniques to genetically engineer microbes used in large-scale manufacturing of the protein. (technologyreview.com)
  • These operations are made possible by recent advances in DNA synthesis and DNA sequencing, providing standardized DNA "parts," modular protein assemblies, and engineering models. (cdc.gov)
  • Expression of the L1 protein using recombinant DNA technology produces noninfectious virus-like particles (VLPs). (cdc.gov)
  • Gene expression in living cells is strictly self-regulated to ensure that the correct amounts of proteins are made at the most appropriate timing and location for maintaining cellular homeostasis. (nature.com)
  • The giver DNA is separated from molecular components of the cell such as proteins, RNA, lipoids and saccharides. (free-essays.us)
  • In transduction, the recombinant molecule is combined with phage caput and tail proteins from one cell to another utilizing bacteriophage vector. (free-essays.us)
  • They then could fish out genes and work forward, biochemically speaking, to discover new proteins that are involved in disease processes. (technologyreview.com)
  • We previously demonstrated that 350 bp of the human rod cGMP phosphodiesterase beta-subunit (beta-PDE) gene promoter are sufficient to direct high levels of gene expression in human Y-79 retinoblastoma cells in vitro. (nih.gov)
  • Photoreceptor-specific reporter gene expression is driven in vivo by a 350-bp segment of the beta-PDE 5' flanking sequence. (nih.gov)
  • This study demonstrates the utility of the human beta-PDE promoter for directing the expression of foreign genes to photoreceptors and suggests that the -297 to +53 beta-PDE 5' flanking region fragment may have important implications for therapeutic gene delivery to the visual cells. (nih.gov)
  • Recent advances have provided a variety of new strategies for delivering DNA and RNA into cells and probing their expression, as well as new clinical applications that rely upon the introduction of genetic material. (cshlpress.com)
  • Gene Transfer: Delivery and Expression of DNA and RNA provides the first comprehensive guide to technical approaches for delivering nucleic acids into cells and organisms and of ensuring (even manipulating) appropriate expression. (cshlpress.com)
  • These include viral and nonviral methods of gene delivery, transgenic approaches, strategies for the regulation of transgene expression, and modification of the host response. (cshlpress.com)
  • To provide a general introduction to the molecular basis of genetics, how genes are maintained from one generation to the next and how their expression is regulated in various systems. (manchester.ac.uk)
  • Students will understand the basis of genome maintenance and gene expression, and appreciate the concept of mutation at the level of the genotype and how this manifests at the phenotypic level. (manchester.ac.uk)
  • Cell-free gene expression systems have been valuable tools for understanding how transcription/translation can be regulated in living cells. (nature.com)
  • Many studies have investigated the determining factors that affect gene expression. (nature.com)
  • for example, the expression level for DNA with 25.7 kbp is 1000-times higher than that for DNA of 1.7 kbp. (nature.com)
  • AFM observation of the DNA conformation indicates that longer DNA takes shrunken conformation with a higher segment density in the reaction mixture for gene expression, in contrast to the stiff conformation of shorter DNA. (nature.com)
  • We propose an underlying mechanism for the favorable effect of longer DNA on gene expression in terms of the enhancement of access of RNA polymerase to the shrunken conformation. (nature.com)
  • It is expected that the enhancement of gene expression efficiency with a shrunken DNA conformation would also be a rather general mechanism in living cellular environments. (nature.com)
  • Gene regulation can occur at any point in gene expression, from the start of the transcription phase to the translation phase. (nature.com)
  • To elucidate the complex mechanism of gene regulation in living cellular systems, it is necessary to closely investigate each phase of gene expression and also to shed light on the cooperative effects between transcription and translation. (nature.com)
  • Although these factors have been studied extensively, little is known about how the length of the DNA template influences gene expression. (nature.com)
  • The principle aim of this project is to isolate TFs genes WRKY, AP2, MYP etc. that specifically regulate the expression of taxol biosynthetic rate limiting gene promoters using transient RNAi. (europa.eu)
  • Based upon the literature, in silico SNP analysis and expression profiling, we were able to identify candidate genes in 2 of these loci: Pla2g7 in lmr1A and Apobec-2 in lmr1B. (rug.nl)
  • This is not surprising given the strong evidence for interaction between myelin and axon gene expression in development and after experimental nerve lesions. (medscape.com)
  • Alpha thalassemia syndromes are caused by deficient expression of 1 or more of the 4 alpha-globin genes on chromosome 16 and are characterized by absent or reduced synthesis of alpha-globin chains. (medscape.com)
  • however, their phenotypic expression may be described in simplified clinical terms related to the number of inherited alpha-globin genes. (medscape.com)
  • Regarding animal models in ASD, many studies focus on gene expression, cortical neuronal migration and cell maturation, and neural network deficits. (bvsalud.org)
  • However, very few studies directly link animal oral communication and gene expression in cortical areas of language. (bvsalud.org)
  • In 1990, as a member of the Physical Mapping Group, Rodriguez gained experience with genomics by participating in the cloning and mapping of the human APOE gene on chromosome 19 at the Lawrence Livermore National Laboratory Shortly thereafter, he created the International Rice Genome Organization, an ad hoc organization of genomics and agriculture experts to develop a strategy for sequencing the rice genome. (wikipedia.org)
  • We used recombinant congenic mouse strains to map asthma susceptibility genes on mouse chromosome 17. (rug.nl)
  • The majority of haemophilia A and B cases follow X chromosome-linked inheritance patterns but haemophilia can also arise from single gene mutations leading to a clotting factor deficiency (1-5). (who.int)
  • The alpha-globin genes are encoded on chromosome 16, and the gamma-, delta-, and beta-globin genes are encoded on chromosome 11. (medscape.com)
  • Alpha-chain genes in duplication on chromosome 16 pairing with non-alpha chains to produce various normal hemoglobins. (medscape.com)
  • Molecular Biology introduces you to the structure and function of molecules, including DNA and RNA, which allow genes to be expressed and be maintained from one generation to the next. (manchester.ac.uk)
  • To exclude the influence of supercoiling, we used linear DNA molecules (PCR amplicons or linearized by restriction enzyme) as a template. (nature.com)
  • The recombinant molecules do non hold covalent bond on rear sugar medieties, the DNA ligase used to seal the sugar molecules and anchor which creates phosphodiester bonds at the functions.DNA ligase widely used in lab and it is derived from the bactriophage T4. (free-essays.us)
  • Synthetic biology is playing an increasing role in the commercial bioeconomy as providers of biological designs, optimized biological molecules, laboratory suppliers of customer-specified DNA, RNA, enzymes and cell-cloning services, and in drug development. (cdc.gov)
  • These principles of biosafety laid the foundation for the NIH Guidelines for Research involving Recombinant or Synthetic Nucleic Acid Molecules, the primary biosafety guidelines in use today [2]. (cdc.gov)
  • The following product was used in this experiment: TERT Recombinant Rabbit Monoclonal Antibody (JF0586) from Thermo Fisher Scientific, catalog # MA5-42464, RRID AB_2911605. (thermofisher.com)
  • Gene therapy and monoclonal antibodies didn't arrive with even a fraction of the hype surrounding embryonic stem cell research. (latimes.com)
  • Advancements in gene editing technologies, especially CRISPR-Cas9, are driving Genetic Engineering Market growth. (emailwire.com)
  • Rapid progress in gene editing offers cost-effective solutions and therapeutic opportunities, driving the global genetic engineering market forward. (emailwire.com)
  • The Genetic Science Learning Center at the University of Utah explains challenges related to gene therapy . (medlineplus.gov)
  • More than 20 different genetic mutations resulting in the functional deletion of both pairs of alpha-globin genes (--/--) have been identified. (medscape.com)
  • There are more than 15 different genetic mutations that result in decreased production of alpha globin, usually through functional deletion of 1 or more of the 4 alpha-globin genes. (medscape.com)
  • Guidelines for the use and safety of genetic engineering techniques or recombinant DNA technology. (who.int)
  • More recently they've also been used to create genes used in gene therapy and genetically modified foods . (thetech.org)
  • Recombinant vaccines are genetically engineered using DNA encoding that stimulates a response in the body, activating the immune system. (fujifilm.com)
  • Parents of a child with a family history of que tienen el gene del antígeno de superficie del virus de la Hepatitis B. El antígeno de superficie de la Hepatitis B (HBsAg) expresado en las genetically engineered Hansenula polymorpha yeast cells having the surface antigen gene of the Hepatitis B virus. (who.int)
  • Hepatitis B vaccine is produced using recombinant DNA technology. (msdmanuals.com)
  • Libyans was 2.18% and 2.20% in 2004 recombinant hepatitis B vaccine at the tive. (who.int)
  • Targeted gene deletion mutants of amlE and amlR were constructed by use of the CRISPR/Cas9 technology. (frontiersin.org)
  • The major concepts and principles underlying recombinant DNA technology will be understood, along with major applications and associated issues relating to the public understanding of science. (manchester.ac.uk)
  • often used as vectors in recombinant DNA technology. (counterbalance.org)
  • I adopted a recombinant DNA technology course and transformed it into molecular biotechnology. (fdu.edu)
  • Forty two years ago, in February 1975, the Asilomar Conference on Recombinant DNA [1] established guiding principles for safe conduct of experiments utilizing recombinant DNA technology, which facilitated the creation of a biotechnology industry in the 1980s. (cdc.gov)
  • The biosafety concerns about synthetic biology and its gene-editing tools are similar to the concerns lodged about recombinant DNA technology when it emerged in mid-1970s. (cdc.gov)
  • While new therapies for haemophilia care have arisen over the past 40 years, today, the usual therapy for haemophilia is factor concentrate, a product routinely developed using recombinant DNA technology (5,6). (who.int)
  • Mutations in this gene have been found in patients with Renpenning syndrome 1 and other syndromes with X-linked cognitive disability. (nih.gov)
  • Gene mutations: missense versus nonsense mutations, insertions, deletions and frameshifts. (manchester.ac.uk)
  • Better understanding of new mutations and the wide range of possible phenotypes led to the development of a new nomenclature proposal, based on the gene and inheritance pattern. (medscape.com)
  • [ 6 ] However, the gene mutations responsible for the different forms of CMT1 are clearly myelin genes. (medscape.com)
  • Mutations and gene deletions causing the thalassemia genotype have arisen independently in different populations but have subsequently propagated by means of natural selection. (medscape.com)
  • From two or three short gene regions, 37% of the strains represented recombinant viruses, multiple infections, or both, which suggests that if whole genome sequences were available, most of these strains would have mosaic genomes. (cdc.gov)
  • Recombinant congenic strains are series of strains which have inherited one or more chromosomal fragments from a donor strain and all other DNA from the background strain. (rug.nl)
  • We then started to map asthma susceptibility in the Lmr series of recombinant congenic and subcongenic strains. (rug.nl)
  • Inoculation with a high dose strains of LMP1 transgenic mice vide a powerful tool in mechanistic of EBV caused a B-cell lymphopro- were established that express LMP1 studies on the role of individual viral liferative disorder in these mice, under the control of the immunoglob- genes in cancer. (who.int)
  • The recombinant DNA techniques were foremost demonstrated in 1972, the parent field of molecular biological science was about entirely an academic field of research. (free-essays.us)
  • As recombinant DNA engineering exposed its practical potency during the 1970s, support for molecular biological science section began to utilize bit by bit more from the private instead than the public sector ( Susan. (free-essays.us)
  • Interest in an undergraduate biochemistry course led me to a Ph.D. program in Biochemistry and Molecular Biology where I researched transcriptional regulation of type I collagen gene in cells from hypertensive fibrotic animal models. (fdu.edu)
  • Interest in signal transduction and hormonal regulation of genes made me adopt an endocrinology course and make it into molecular endocrinology. (fdu.edu)
  • LMO1 Human Recombinant produced in E.Coli is a single, non-glycosylated polypeptide chain containing 179 amino acids (1-156 a.a) and having a molecular mass of 20.2kDa. (prospecbio.com)
  • While the transfer of already existing genes from one cell to another characterized an earlier phase of the field of biotechnology, synthetic biology involves the design, assembly, synthesis, or manufacture of new genomes, biological pathways, devices or organisms not found in nature. (cdc.gov)
  • DNA is a double stranded molecule. (thetech.org)
  • The two DNA molecule sites can crossbreed to each other through their reconciliation gluey ends to organize a recombinant molecule. (free-essays.us)
  • In transmutation, recombinant DNA molecule enters into bacteriums cells. (free-essays.us)
  • FUJIFILM Diosynth Biotechnologies' labs support the creation of gene therapy drugs and the vectors that deliver them. (fujifilm.com)
  • FUJIFILM Diosynth Biotechnologies works with a variety of viral vectors and cell culture platforms to help develop the manufacturing process, leading to commercial production for gene therapy products. (fujifilm.com)
  • The biosynthesis genes are encoded by the acb gene cluster, which was identified in 1999 by Stratmann et al. (frontiersin.org)
  • The attractive features of this study, examining the elicitor-induced regulator of selected TFs genes to see whether their mRNA levels correlate with fungal taxol biosynthesis. (europa.eu)
  • Experiments involving the deliberate formation of synthetic or recombinant DNA containing genes for the biosynthesis of select toxins lethal for vertebrates at an LD 50 <100 ng/kg body weight (ie, nucleic acids that encode select toxins). (cdc.gov)
  • This time, we succeeded to develop the recombinant retro-virus vector expressiog EGFR antisense oligonucleotide, and we could inhibit the growth of the malignant glioma cell line using this retro-virus vector. (nii.ac.jp)
  • Recombinant adenovirus type 5 (rAd) has been used as a vaccine platform against many infectious diseases and has been shown to be an effective vaccine vector. (mdpi.com)
  • FUJIFILM Diosynth Biotechnologies' high-tech labs build APIs for different types of vaccines, including viral vector and recombinant DNA vaccines. (fujifilm.com)
  • Genes that regulate both the synthesis and the structure of different globins are organized into 2 separate clusters. (medscape.com)
  • Your second year builds on this knowledge and covers areas such as gene regulation, cell biology and metabolism. (kent.ac.uk)
  • Insulin as first commercial product from gene transfer. (greenfacts.org)
  • One of the biggest mileposts, readying of HUMAN INSULIN utilizing recombinant DNA engineering. (free-essays.us)
  • Understanding gene function and regulation requires rigorous testing in live cells and organisms. (cshlpress.com)
  • A second capability of synthetic biology involves the re-design of existing genes, cells or organisms for the purpose of gene therapy. (cdc.gov)
  • Modification of existing genes in living animal and human cells is enabled by engineered nucleases such as meganucleases, zinc finger nucleases, transcription activator-like effector-based nucleases, and the CRISPR-Cas system. (cdc.gov)
  • Tiomersal es agregado como surface antigen (HBsAg) expressed in the cells of Hansenula polymorpha is purified through several chemical steps using recombinant regarding appropriate medical care in the unlikely event of a seizure. (who.int)
  • Although AmlR apparently is a local transcriptional regulator of the aml operon, the Δ amlR strain shows severe growth inhibitions on glucose and - concomitantly - differential transcription of several genes of various functional classes. (frontiersin.org)
  • This research led me to post-doctoral fellowships predominantly on the extracellular matrix genes methylation, transcription, cloning, and photomedicine at Boston University Medical Center, Massachusetts General Hospital, and NYU Medical Center. (fdu.edu)
  • The protocol, or plan, for each clinical trial is then reviewed by the NIH Recombinant DNA Advisory Committee (RAC) to determine whether it raises medical, ethical, or safety issues that warrant further discussion at a RAC public meeting. (medlineplus.gov)
  • Scientists first started making synthetic genes by cutting and pasting parts of different genes together. (thetech.org)
  • Designing and making synthetic genes has been done for decades, although the technologies have gotten better with time. (thetech.org)
  • The second is creating a synthetic gene directly from DNA. (thetech.org)
  • One way to make a synthetic gene is to recombine these instructions. (thetech.org)
  • Progress in this branch of synthetic biology has yielded remarkable therapeutic advances in gene therapy well beyond the achievements of conventional drugs and biologic agents. (cdc.gov)
  • EC 2.1.1.6) was determined from rat cDNA and genomic libraries were screened with DNA probes and specific antiserum. (lu.se)
  • A putative acylphosphatase gene (PhAcP) was cloned from the genomic DNA of Pyrococcus horikoshii and was expressed in Escherichia coli. (rcsb.org)
  • Structure of chromosomes: DNA packaging, features of metaphase chromosomes. (manchester.ac.uk)
  • This vaccine cannot cause hepatitis B virus infection because no potentially infectious viral DNA or complete viral particles are produced during this process. (msdmanuals.com)
  • Employing the DNA blot analysis only one COMT-encoding gene was found in the rat genome. (lu.se)
  • Recombinant DNA was the stem cell-style biomedical panacea of the 1960s and early 1970s. (latimes.com)
  • Neither a gene encoding a maltose phosphorylase (MalP) nor MalP enzyme activity were detected in the wild type. (frontiersin.org)
  • So if you know the DNA letters of your gene, then you can use the right enzyme to cut it in the right place. (thetech.org)
  • The complementary DNA is prepared from messenger RNA of retroviruses utilizing particular enzyme contrary RNA polymerase. (free-essays.us)
  • A construct containing the bacterial LacZ gene driven by a fragment of the beta-PDE 5' flanking region (-297 to +53) was used to generate transgenic mice. (nih.gov)
  • We propose that the HIV epidemic was established in Central Africa by the early 1980s and that some recombinant viruses most likely seeded the early global epidemic. (cdc.gov)
  • The group M viruses can be subdivided into 9 subtypes, A-D, F-H, J, and K, and at least 14 circulating recombinant forms (CRFs) ( http://hiv-web.lanl.gov ). (cdc.gov)
  • In the presence of DNAβ01, these viruses induced mild leaf curl symptoms in tomato plants. (apsnet.org)
  • A plasmid containing the gene for hepatitis B surface antigen (HBsAg) is inserted into common baker's yeast, which then produces HBsAg. (msdmanuals.com)
  • These enzymes are like scissors that chop up double stranded DNA. (thetech.org)
  • Small circles of double-stranded DNA found in some bacteria. (counterbalance.org)
  • LMO1 encodes a transcriptional regulator which contains two cysteine-rich LIM domains but lacks a DNA-binding domain. (prospecbio.com)
  • The object of our study aimed to review and to correlate genes involved in ASD and those related to ultrasonic communication in animal model studies of language-based social behavior at the PubMed database. (bvsalud.org)
  • Replication of genomes: origins of replication, control of DNA replication. (manchester.ac.uk)
  • Two begomoviruses (Java virus-1 and Java virus-2), two satellite DNAs (DNAβ01 and DNAβ02), and a recombinant DNA (recDNA) were cloned from a single tomato plant from Indonesia with leaf curl symptoms, and the role of these satellite DNAs in the etiology of begomovirus disease was investigated. (apsnet.org)
  • Here we report the effect of the length of linearized reporter DNAs encoding the firefly luciferase gene so as to exclude the influence of supercoiling. (nature.com)
  • Lab designed genes are not only used by scientists to study biology. (thetech.org)
  • Cloning of malaria parasite DNA / by Nednapis Tirawanchai. (who.int)
  • Gene regions representing all the HIV-1 group M clades and unclassifiable sequences were found. (cdc.gov)
  • New DNA sequences can be made by cutting and pasting other pieces of DNA together. (thetech.org)
  • But different enzymes cut the DNA at different specific sequences. (thetech.org)
  • The first lab-designed gene was made thirty-five years ago in 1972. (thetech.org)
  • This procedure of section is called a DNA palindrome which explains the both strands have same nucleotide sequence but antiparallel placement. (free-essays.us)
  • The earliest studies showed that gene therapy could have very serious health risks, such as toxicity, inflammation, and cancer. (medlineplus.gov)
  • Advent of gene transfer through recombinant DNA techniques. (greenfacts.org)
  • NIH provides guidelines for investigators and institutions (such as universities and hospitals) to follow when conducting clinical trials with gene therapy. (medlineplus.gov)
  • An Institutional Review Board (IRB) and an Institutional Biosafety Committee (IBC) must approve each gene therapy clinical trial before it can be carried out. (medlineplus.gov)
  • Publications] Noriaki Sugawa, Satoshi Ueda: 'Gene therapy using antisense DNA' Clinical Neuroscience. (nii.ac.jp)
  • Gene therapy treats or prevents disease by replacing a faulty gene, inactivating a mutated gene, or adding a new gene. (fujifilm.com)
  • Gene therapy drugs can be challenging to produce, and many are under clinical trials. (fujifilm.com)
  • Is gene therapy safe? (medlineplus.gov)
  • The first gene therapy trial was run more than thirty years ago. (medlineplus.gov)
  • however, medical researchers, institutions, and regulatory agencies are working to ensure that gene therapy research, clinical trials, and approved treatments are as safe as possible. (medlineplus.gov)
  • The U.S. Food and Drug Administration (FDA) regulates all gene therapy products in the United States and oversees research in this area. (medlineplus.gov)
  • The National Institutes of Health (NIH) also plays an important role in ensuring the safety of gene therapy research. (medlineplus.gov)
  • Multiple levels of evaluation and oversight ensure that safety concerns are a top priority in the planning and carrying out of gene therapy research. (medlineplus.gov)
  • I branched to include more genes as targets, and more diseases and pharmaceuticals or everyday actives, to understand the mechanisms of effects. (fdu.edu)
  • Soon after its approval by the NIH, pBR322 was used to clone and express the first chemically synthesized gene for the human peptide hormone, somatostatin. (wikipedia.org)
  • From DNA to RNA: the structure and function of the gene, promoters and terminators. (manchester.ac.uk)
  • This project offer de nova interactions between the TFs and the TB gene promoters, which results of DNA binding cis-elements. (europa.eu)
  • Forty years ago, when transferring genes from one organism to another was first becoming a standard research technique, scientists naturally began to worry about its potential hazards. (i-sis.org.uk)
  • This precise and efficient tool enables targeted DNA modifications, accelerating research and applications in fields like agriculture and medicine. (emailwire.com)
  • Recently the research workers developed complementary DNA from chemical procedure. (free-essays.us)
  • Within the retina, the reporter gene was specifically expressed in photoreceptors, consistent with the localization of endogenous beta-PDE. (nih.gov)
  • Thalassemia is one of the world's most common single-gene disorders. (medscape.com)