-------------------------------------------------------------------------------------- |.gov |.edu |.org |.com || ------Reading----------------Writing-------------Arithmetic-------

Slides:



Advertisements
Similar presentations
1. 2 Key Applications of Genetic and Genomic Testing (slide 1 of 2) Diagnosis of Disease: Whereby genetic or genomic tests are used to screen a patient.
Advertisements

Reproductive Technology, Genetic Testing, and Gene Therapy
Growth and development Samir Najjar M.D Professor, Department of Pediatrics.
59%23% 4% Figure 1. Distribution of Health Insurance Coverage, New York State, 2004– million people Note: Data include all persons below age 65.
NS 1300 Emergence of Modern Science Biotechnology.
Abbey Deutschman Brittany Prentice Brittany Prentice Colby Peters Colby Peters.
Positional Cloning LOD Sib pairs Chromosome Region Association Study Genetics Genomics Physical Mapping/ Sequencing Candidate Gene Selection/ Polymorphism.
Genetic Engineering.
Genetic Engineering: Some Basic Concepts. DNA: The Information Carrier.
5.2.3 Gene Therapy explain the term gene therapy; explain the differences between somatic cell gene therapy and germ line cell gene therapy; discuss the.
Clinical Genetics Cytogenetics Molecular Genetics National Centre for Medical Genetics.
Careers in Biotechnology
DESIGNER BABIES. What?  Definition: The term used to define the genetic engineering of an embryo’s genes and genome in order to specify the genes of.
GENETIC ENGINEERING Todd Sachs. Is DNA modification acceptable?
Smoking Cessation for Pregnancy and Beyond: Virtual Clinic Companion Slides Catherine A. Powers, EdD, LSW PACE – Tobacco Prevention and Cessation Education.
Genetic Disorders, Blood Typing, and Genetic Engineering.
KEY CONCEPT Genetics provides a basis for new medical treatments.
Gene Therapy Correcting defective genes. Definition & history  Normal gene inserted into the genome to replace non-functional gene  Trials began in.
Sally Freese Family and Consumer Science
Application of gene technology
MODERN GENETICS USES DNA TECHNOLOGY
Unit 5 – Public Health Chronic Diseases
Epigenome 1. 2 Background: GWAS Genome-Wide Association Studies 3.
Genetic technology. Some terminology Genetic engineering –Direct manipulation of genes for practical purposes Biotechnology –Manipulation of organisms.
Exploring Technology. Solving Real-World Problems Many issues in our world are related to biology Biologists are working to try to solve these problems.
Treatment of short stature M. Hashemipour MD Pediatric Endocrinologist Isfahan University of Medical Sciences 1.
Gene Therapy Trials for PID:A Nursing Perspective Jin Hua Xu-Bayford Clinical Nurse Specialist Gene Therapy The child first and.
Thanks to:.gov ||.edu ||.org ||.com || Read = = = = = = = = I/O = = = = = = = Write 1 Information Technolgy Future of Medicine 11:13-11:20 AM 5-Jun-2012.
Genetic Issues We are at the dawn of a new age in medicine Genetically engineered therapy will personalize medicine Diagnosis will precede disease This.
Bringing Genomics Home Your DNA: A Blueprint for Better Health Dr. Brad Popovich Chief Scientific Officer Genome British Columbia March 24, 2015 / Vancouver,
Why ? And what are the current alternatives ? The Francis Crick Institute, London, UK Moderator: Robin Lovell-Badge Applications of Gene Editing Technology:
KEY CONCEPT Genetics provides a basis for new medical _____________.
What is gene therapy? Do now: In your own words,
Case studies in gene editing… George Q. Daley, MD, PhD Director, Stem Cell Transplantation Program Boston Children’s Hospital/ Dana Farber Cancer Institute.
1 Jin-Soo Kim Dept. of Chemistry, Seoul National Univ. Center for Genome Engineering, Institute for Basic Science Genome Editing: Challenges and Opportunities.
The French Perspective The law 1994 Civil code: Art
Gene editing in embryos and germ line
Introduction.
HBV (chronic) HIV ~400 million chronically infected
Massachusetts General Hospital/ Harvard Medical School
Gene Therapy In and Around the Germline Kyle Orwig Magee-Womens Research Institute University of Pittsburgh School of Medicine International Summit on.
Cloning.
Ornithine transcarbamylase deficiency: the story of Jesse Gelsinger.
Examples of Completed Genomes :. IV. Human Gene Therapy Seeks to treat disease by altering an afflicted person’s genes. A mutant gene may be replaced.
Cri-du-chat Syndrome By: Maddy Gordon. What is Cri-du-chat syndrome? Cri-du-chat syndrome is a rare chromosomal condition that results when a piece of.
INTERPRETING GENETIC MUTATIONAL DATA FOR CLINICAL ONCOLOGY Ben Ho Park, M.D., Ph.D. Associate Professor of Oncology Johns Hopkins University May 2014.
Genetics of Cardiomyopathy Affairs of the Heart: Living with Inherited Cardiomyopathy February 20, 2016 Kyla Dunn, MS, LCGC Cardiovascular Genetic Counselor.
Recent Advances in Genomic Science Julian Sampson Institute of Medical Genetics, Cardiff.
Next generation genomics: translation into clinically useful applications in health care Prof.dr. Martina Cornel
How did the pig on pg. 342 become ‘glowing’? How does this relate to the transformation lab?
What do we need to know to become stem cell literate?
Brenda Wilson Institute for Science, Society and Policy
BioTech and Health Career opportunities Phenylketonuria (PKU)
Genetic Engineering Nate, Belle, and Kaitlin.
New Approaches to Cancer Susceptibility Testing
AKA….Biotechnology/ Genetic Engineering
WELCOME to Bio 230: Genetics Dr. Erin Gestl Jan 24 Pages 1-28.
What makes a mutant?.
Correcting defective genes
Class: ENSC S-135 Biochemical Engineering and Synthetic Life
KEY CONCEPT Genetics provides a basis for new medical treatments.
KEY CONCEPT Genetics provides a basis for new medical treatments.
KEY CONCEPT Genetics provides a basis for new medical treatments.
Activity 1: Causes of Human Population Growth
KEY CONCEPT Genetics provides a basis for new medical treatments.
KEY CONCEPT Genetics provides a basis for new medical treatments.
Activity 1: Causes of Human Population Growth
changes that occur to genes that result in medical conditions.
KEY CONCEPT Genetics provides a basis for new medical treatments.
Presentation transcript:

|.gov |.edu |.org |.com || Reading Writing Arithmetic Human Germline Modification 1-Dec PM NAS LSRF NHGRI NIGMS Azco Oppenheimer Foundation PersonalGenomes.org

Meth lab seizures per county DEA's National Clandestine Laboratory Register Adverse Reactions for Dietary Supplements Ineffective & unsafe, yet widespread

Safe & effective relative to alternatives, yet concerns persist 1796 Vaccination vs smallpox, polio... GMO Golden rice vs 1M deaths/yr Test-tube babies vs infertility Random vs engineered

Compelling medical applications of germline therapies 1) Mitochondrial diseases 2) Families in which post-natal remedies are not optimal and both parents are fully afflicted (20% of marriages involve close relatives) 3) Treating & screening single germ cells is safer than treating millions of somatic cells, since each cell adds to the collective risk of cancer. 4) PGD-IVF is not acceptable for medical or personal reasons. 5) Complex diseases with single-target solutions, which work better when applied earlier.

Nuclease Specificity 100-fold: Theoretical & empirical searches Mali et al., Cong et al. (Church & Zhang labs) Science 3-Jan fold: Paired nickases. Mali, et al, Ran et al (Church, Zhang labs). Nat Biotechnol. 1-Aug Cell 12-Sep-2013 <5,000-fold: Truncated guide RNAs. Fu, et al. (Joung lab). Nat Biotechnol. 26-Jan-2014 >100-fold: RNA-guided FokI nucleases (Joung & Liu labs) Nat Biotechnol. 25-Apr-2014 >25-fold: Cas9 charge Slaymaker (Zhang lab) Science 1-Dec-2015

Efficiency & specificity goals? Off-target: 10,000 to 10 events per million cells Spontaneous error rate = 0.1 to 10 per cell. Clonal cells, e.g. spermatogonial stem cells (SSC) Increase efficiency from 5-50% to 100%. (& Specificity.) Analysis: BLESS, Guide-Seq, Digenome-seq, HTGTS, IDLV, whole genome, targeted seq. Haploinsufficient = ~3000 journals.plos.org/plosgenetics/article?id= /journal.pgen bioinfo.mc.vanderbilt.edu/TSGene #cells: pubmed/

We are not limited by natural variants or complexity: GH therapies for non-GH alleles Turner syndrome Chronic renal failure Prader–Willi syndrome Intrauterine growth retardation Idiopathic short stature AIDS Muscle wasting Also, Not limited to gene therapies: genetic counseling, gene drives, xenotransplantation

Enhancement via somatic gene therapies Somatic protocols can spread more rapidly than germline modifications, since only 1% of the population per year (that is births) are of the correct age for germline impact, while somatic therapy is applicable at any age. Pathogen resistance CCR5, FUT2 Aging GFD11-MSTN, TERT-CDKN2A-TP53 Cognitive (ASD/AD) NGF, NEU1, GRIN2B, PDE4B

Equitable access to technology? Super-exponential technologies. Encouraging innovation. $1k 2015 Not 6 decades but 6 years Moore’s “Law” arep.med.harvard.edu/gmc/genome_services.html $ From $3 billion to $1000