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GETTING STARTED INTRODUCTION TO GENETICS,What is a gene. Before we describe an approach to gene therapy in Huntington s disease and the. opportunity to participate in a clinical trial we want to review some important terms Genes make up the instruction manual used to make you into. Here are a few words that you need to understand before reading this brochure the person you are today 1 Did you know that humans have about. 30 000 genes 2,TERM DEFINITION, Each cell within your body contains 2 sets of chromosomes or. The cells of your body there are trillions of them are considered the building. tightly packed bundles of genes These chromosomes are passed. Cell blocks of all living things They provide structure and function to all parts of your. body Cells contain DNA down from generation to generation You inherit 1 set of chromo. somes from your mother and the other set from your father 1. Chromosome A bundle of DNA found within the nucleus of cells. Material found in the nucleus of cells that contains instructions to direct. the cell s activity Your DNA contains a unique code that makes you you. A section of DNA that provides the instructions for the cell to make Gene. a certain protein, Gene therapy A variety of medical treatments that use genetic material to treat diseases. Genetic disorder An illness caused by 1 or more genes that are not working properly. Cell Chromosome DNA,Inherit To receive genetic material from parents. Messenger RNA represents an intermediate step in the protein making Chromosomes are tightly packed bundles of DNA which provide cells. Messenger RNA, process and contains the instructions to direct the cell s activity Typically instructions to make proteins A gene is a segment of DNA 1.
mRNA DNA is converted to messenger RNA which is translated to proteins. Mutant protein A change in the structure of an encoded protein that alters the function. GENETIC DISORDERS, The core of the cell that contains the DNA and provides instructions about. how the cell should grow mature divide or die,What is a genetic disorder. Genetic disorders result when an error or a change in a gene leads. Proteins provide structure to the parts of the body and serve a variety of functions. Protein to a problem with the making of protein and causes a disease 3 Most. examples of types of proteins include antibodies enzymes and hormones. of the time when we think about a genetic disorder we re talking. Definitions are based on the literature and the following sources about a disorder you inherit or a congenital disorder. American Society of Gene and Cell Therapy https www asgct org education glossary. US National Genetics Home Reference https ghr nlm nih gov primer. TYPES OF GENE THERAPY What is the approach to gene therapy. What is gene therapy for Huntington s disease, Gene therapy is the method of using genetic material to treat or prevent Gene regulation is the type of gene therapy being applied in the uniQure. disease 4 Gene therapy is being developed for the treatment of serious Huntington s disease clinical trial It is designed to reduce the production. genetic disorders such as Huntington s disease 5 of huntingtin protein Build up of mutant huntingtin protein is thought. to cause the symptoms of Huntington s disease 8, Are there other gene therapies approved In order to reduce the levels of mutant huntingtin protein multiple. approaches are being studied that target different points of the protein. There are approved gene therapies in the United States One ZOLGENSMA. making process, is for people with spinal muscular atrophy an inherited genetic disorder.
that affects special cells within the spinal cord 6 The other LUXTURNA DNA messenger RNA Protein. is for people with inherited retinal diseases 7 Both medicines use the same. delivery system AAV that is used in the gene therapy for Huntington s disease. gets converted to which gets translated to, In the uniQure Huntington s disease clinical trial gene therapy is targeting. messenger RNA which plays a role in how proteins get made The gene. therapy allows cells to produce blocking messenger RNA that interferes. with Huntington s disease messenger RNA preventing the production. of huntingtin protein 8,What are the goals of gene therapy. for Huntington s disease, n Reduce the production of mutant huntingtin protein throughout the brain. n Preserve the function of brain cells, n Prevent or delay the progression of the symptoms of Huntington s disease. n Establish long term multiyear benefit from a 1 time procedure. n Maintain the patient s quality of life,UNDERSTANDING GENE THERAPY.
Follow along the 3 steps shown here for easy understanding. Continuing to make, 1 Administering gene therapy 2 Entering the brain cell 3 blocking messenger RNA. Caudate nucleus Therapeutic,Putamen gene,Brain cell. Illustrations by Virginia Ferrante Iqbal, During a 1 time procedure the gene therapy will The gene therapy delivers instructions to make The gene therapy may stay in brain. be administered into the caudate nucleus and blocking messenger RNA that interferes with cells and continue to make blocking. putamen together known as the striatum the areas Huntington s disease messenger RNA preventing messenger RNA preventing the production. most affected by mutant huntingtin protein 8 the production of huntingtin protein 8 of huntingtin protein over time 8. RESOURCES FOR THE,HUNTINGTON S DISEASE COMMUNITY, UNIQURE GENE Huntington s Disease Society of America HDSA. https hdsa org,THERAPY APPROACH Helpline 800 345 HDSA 4372.
REFERENCES,1 Introduction to Genomics Genome gov https. IN HUNTINGTON S National Office 212 242 1968 www genome gov About Genomics Introduction to. Genomics Accessed December 18 2019,Email HDSAinfo HDSA org. DISEASE 2 Human Genome Project FAQ Genome gov https www. genome gov human genome project Completion FAQ, Huntington s Disease Youth Organization HDYO Accessed December 18 2019. uniQure is studying a new gene https en hdyo org 3 Genetics Home Reference Do all gene mutations affect. therapy in clinical trials to assess Email support hdyo org health and development Genetics Home Reference. the safety tolerability and efficacy https ghr nlm nih gov primer mutationsanddisorders. Postal address neutralmutations Accessed December 18 2019. of a 1 time treatment in adults HDYO 4 Genetics Home Reference What is gene therapy. with Huntington s disease The PO Box 6371 https ghr nlm nih gov primer therapy genetherapy. Accessed December 18 2019,gene therapy is intended to Delray Beach FL 33482. 5 Pfalzer A Details emerge of first Huntington s disease. reduce the production of mutant United States gene therapy clinical trial HD Buzz https en hdbuzz. huntingtin protein which may net 274 Accessed December 18 2019. For AMT 130 Clinical Trial Information Visit, slow the progression of Huntington s 6 Zolgensma R onasemnogene abeparvovec xioi Pre.
Go to https clinicaltrials gov and search AMT 130 and uniQure scribing information AveXis Inc Bannockburn IL 2019. disease 5 8 For more information 7 Luxturna voretigene neparvovec rzyl Prescribing infor. about Huntington s Disease please Comments or Questions mation Spark Therapeutics Inc Philadelphia PA 2017. talk to your physician amt130 clinical trials uniqure com or dial 1 339 970 7081 8 uniQure Data on file 2019. About uniQure,uniQure delivers on the promise,of gene therapy single treatments. with potentially curative results The,company is developing several gene. therapies for the treatment of patients,with liver metabolic central nervous. Gene therapy A variety of medical treatments that use genetic material to treat diseases Genetic disorder An illness caused by 1 or more genes that are not working properly Inherit To receive genetic material from parents Messenger RNA mRNA Messenger RNA represents an intermediate step in the protein making process and contains the instructions to direct the cell s activity Typically

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