Genes and Gene Therapy (Print Version)

Genes are the building blocks of inheritance. Passed from parent to child, they contain instructions for making proteins. If genes don't produce the right proteins or don't produce them correctly, a child can have a genetic disorder.

Gene therapy is an experimental technique that uses genes to treat or prevent disease. The most common form of gene therapy involves inserting a normal gene to replace an abnormal gene. Other approaches include

Swapping an abnormal gene for a normal one
Repairing an abnormal gene
Altering the degree to which a gene is turned on or off

Although there is much hope for gene therapy, it is still experimental.

Genetics Home Reference

Start Here

Frequently Asked Questions about Genetic and Genomic Science(National Human Genome Research Institute) - http://www.genome.gov/19016904
Genetics Home Reference: What is gene therapy?(National Library of Medicine) - http://ghr.nlm.nih.gov/handbook/therapy/genetherapy
JAMA Patient Page: Genetics: The Basics(American Medical Association) - http://jama.ama-assn.org/cgi/reprint/299/11/1388.pdf
Also available in Spanish http://jama.ama-assn.org/cgi/data/299/11/1388/DC1/1

Overviews

From Genes to Personalized Medicine(National Institutes of Health) - Links to PDF - http://www.nih.gov/about/researchresultsforthepublic/Genes_PersonalizedMed.pdf
Gene Therapy(Mayo Foundation for Medical Education and Research) - http://www.mayoclinic.com/print/gene-therapy/MY00105/METHOD=print&DSECTION=all
Gene Therapy(Dept. of Energy) - http://www.medem.com/medlb/article_detaillb.cfm?article_ID=ZZZBLSC7W7C&sub_cat=203
Genes -- What We Knew, Know, and Hope to Learn(National Institute of General Medical Sciences) - http://www.nigms.nih.gov/Publications/FactSheet_Genes.htm
New Genetics(National Institute of General Medical Sciences) - http://publications.nigms.nih.gov/thenewgenetics/

Latest News

Overeating in the Genes for Some Kids, Study Says(01/12/2009, Reuters Health) - http://www.nlm.nih.gov/medlineplus/news/fullstory_73697.html
Gene Fusions May Drive Certain Cancers(01/12/2009, HealthDay) - http://www.nlm.nih.gov/medlineplus/news/fullstory_73716.html
Gene Variant May Hike Women's Risk of Alzheimer's(01/12/2009, HealthDay) - http://www.nlm.nih.gov/medlineplus/news/fullstory_73717.html
More News on Genes and Gene Therapy http://www.nlm.nih.gov/medlineplus/alphanews_g.html#genesandgenetherapy

Related Issues

FDA 101: Human Gene Therapy(Food and Drug Administration) - http://www.fda.gov/consumer/updates/genetherapy022608.html
Gene Therapy for Cancer: Questions and Answers(National Cancer Institute) - http://www.cancer.gov/cancertopics/factsheet/Therapy/gene
Genetics Home Reference(National Library of Medicine) - http://ghr.nlm.nih.gov/
How FDA Advances Personalized Medicine(Food and Drug Administration) - http://www.fda.gov/consumer/features/personalmed090607.html
Human Genetic Variation(National Institute of General Medical Sciences) - http://www.nigms.nih.gov/Publications/Factsheet_GeneticVariation.htm
Pharmacogenomics: When Drug Treatment Becomes Personalized Medicine(Mayo Foundation for Medical Education and Research) - http://www.mayoclinic.com/print/personalized-medicine/CA00078/METHOD=print
Proteomics(American Medical Association) - http://www.ama-assn.org/ama/pub/category/3668.html

Games

Play the DNA - The Double Helix Game(Nobel Foundation) - http://nobelprize.org/educational_games/medicine/dna_double_helix/
Play the Genetic Code Game(Nobel Foundation) - http://nobelprize.org/educational_games/medicine/gene-code/

Anatomy/Physiology

Gene-Environment Interaction Fact Sheet(Centers for Disease Control and Prevention) - http://www.cdc.gov/genomics/training/perspectives/factshts/geneenviro.htm

Clinical Trials

ClinicalTrials.gov: Gene Therapy(National Institutes of Health) - http://clinicaltrials.gov/search/open/intervention=%22gene+therapy%22

Research

All about the Human Genome Project (HGP)(National Human Genome Research Institute) - http://www.genome.gov/10001772
Cell-Signaling System Helps Explain How Skin Develops into Different Types(National Institute of Arthritis and Musculoskeletal and Skin Diseases) - http://www.niams.nih.gov/News_and_Events/Spotlight_on_Research/2008/Cell_Signaling_Skin_Devel.asp
Genes or Environment? Epigenetics Sheds Light on Debate(National Institutes of Health) - http://newsinhealth.nih.gov/2006/February/docs/01features_02.htm
Genes, Behavior, the Environment and Health(National Institutes of Health) - Links to PDF - http://www.nih.gov/about/researchresultsforthepublic/GenesandBehavior.pdf
Matching Genes and Vitamins: A Personalized Plan May Be in Your Future(National Institutes of Health) - http://newsinhealth.nih.gov/newversion/2008/August/feature2.htm
Medicines for You: Studying How Your Genes Can Make a Difference(National Institute of General Medical Sciences) - http://publications.nigms.nih.gov/medsforyou/
Also available in Spanish http://publications.nigms.nih.gov/medsforyou/index_esp.html
Pharmacogenetics(National Institute of General Medical Sciences) - http://www.nigms.nih.gov/Initiatives/PGRN/Background/FactSheet.htm

Journal Articles

References and abstracts from MEDLINE/PubMed (National Library of Medicine)

Article: Emerging technologies and fourth generation issues in cartilage repair. - http://www.ncbi.nlm.nih.gov/sites/entrez?cmd=Retrieve&db=pubmed&dopt=AbstractPlus&list_uids=19011557&tool=MedlinePlus
Article: Novel jet-injection technology for nonviral intratumoral gene transfer in patients... - http://www.ncbi.nlm.nih.gov/sites/entrez?cmd=Retrieve&db=pubmed&dopt=AbstractPlus&list_uids=19010873&tool=MedlinePlus
Article: Gene and cell delivery to the degenerated striatum: status of... - http://www.ncbi.nlm.nih.gov/sites/entrez?cmd=Retrieve&db=pubmed&dopt=AbstractPlus&list_uids=18981876&tool=MedlinePlus
Genes and Gene Therapy -- see more articles - http://www.ncbi.nlm.nih.gov/sites/entrez?cmd=search&db=pubmed&term=gene+therapy[majr]+AND+humans[mh]+AND+english[la]+AND+(review[pt]+OR+guideline[pt]+OR+clinical+trial[pt]+OR+jsubsetk[text]+OR+jsubsetaim[text]+OR+jsubsetn[text]+OR+patient+education+handout[pt]+NOT+(letter[pt]+OR+editorial[pt]+OR+case+reports[pt]))&doptcmdl=summary&cmd_current=Limits&pmfilter_EDatLimit=last+1+Year&tool=MedlinePlus
Gene mapping -- see more articles - http://www.ncbi.nlm.nih.gov/sites/entrez?cmd=search&db=pubmed&term=chromosome+mapping[majr]+OR+genome,+human[majr]+AND+humans[mh]+AND+english[la]+AND+(jsubsetaim[text]+OR+jsubsetk[text]+OR+jsubsetn[text]+OR+systematic[sb]+OR+patient+education+handout[pt])+NOT+(letter[pt]+OR+editorial[pt])&doptcmdl=summary&cmd_current=Limits&pmfilter_EDatLimit=last+1+Year&tool=MedlinePlus

Dictionaries/Glossaries

"-OMICS" Glossary: A Selection of Important Terms and Concepts Defined(Food and Drug Administration) - http://www.fda.gov/fdac/features/2005/605_glossary.html
Talking Glossary of Genetic Terms(National Human Genome Research Institute) - http://www.genome.gov/glossary.cfm
Also available in Spanish http://www.genome.gov/sglossary.cfm

Directories

Laboratory Directory-GeneReviews(University of Washington) - http://www.genetests.org/servlet/access?id=8888891&key=xW0l31Z2a4hTc&fcn=y&fw=XPF9&filename=/labsearch/searchdztest.html

Organizations

GeneTests(University of Washington) - http://www.genetests.org/
National Human Genome Research Institute - http://www.genome.gov/

Newsletters/Print Publications

Genomics & Health Weekly Update(Centers for Disease Control and Prevention) - http://www.cdc.gov/genomics/update/current.htm

Law and Policy

Issues in Genetics and Health(National Human Genome Research Institute) - http://www.genome.gov/10001740
Policy and Legislation Database(National Human Genome Research Institute) - http://www.genome.gov/PolicyEthics/LegDatabase/pubsearch.cfm

Children

Gene Therapy and Children(Nemours Foundation) - http://kidshealth.org/parent/system/medical/gene_therapy.html
What Is a Gene?(Nemours Foundation) - http://kidshealth.org/kid/talk/qa/what_is_gene.html
You and Your Genes: Making It in a Tough Environment(National Institute of Environmental Health Sciences) - Links to PDF - http://www.niehs.nih.gov/health/scied/documents/You-YourGenes.pdf

Teenagers

Basics on Genes and Genetic Disorders(Nemours Foundation) - http://kidshealth.org/teen/your_body/health_basics/genes_genetic_disorders.html
Also available in Spanish http://kidshealth.org/teen/en_espanol/cuerpo/genes_genetic_disorders_esp.html

You may also be interested in these MedlinePlus related pages:

Genetic Disorders - http://www.nlm.nih.gov/medlineplus/geneticdisorders.html
Genetic Testing - http://www.nlm.nih.gov/medlineplus/genetictesting.html
Genetics/Birth Defects - http://www.nlm.nih.gov/medlineplus/geneticsbirthdefects.html

The primary NIH organization for research on Genes and Gene Therapy is the National Human Genome Research Institute - http://www.genome.gov/