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
- 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
- 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/
- Vitamin D Receptor Gene Variant Tied to Melanoma(09/22/2008, HealthDay) - http://www.nlm.nih.gov/medlineplus/news/fullstory_69561.html
- Healthy Lifestyle Raises Beneficial Enzyme(09/16/2008, Reuters Health) - http://www.nlm.nih.gov/medlineplus/news/fullstory_69316.html
- Gene Variant Raises Skin Cancer Risk(09/15/2008, Reuters Health) - http://www.nlm.nih.gov/medlineplus/news/fullstory_69263.html
- More News on Genes and Gene Therapy http://www.nlm.nih.gov/medlineplus/alphanews_g.html#genesandgenetherapy
- 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/
- Genomics and Medical Devices: A New Paradigm for Health Care(Food and Drug Administration) - http://www.fda.gov/fdac/features/2005/605_devices.html
- Genomics and Personalized Medicine(Food and Drug Administration) - http://www.fda.gov/fdac/features/2005/605_genomics.html
- 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
- Metabolomics: Working toward Personalized Medicine(Food and Drug Administration) - http://www.fda.gov/fdac/features/2005/605_metabolomics.html
- 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: Moving Beyond the Human Genome(Food and Drug Administration) - http://www.fda.gov/fdac/features/2005/605_proteomics.html
- Play the DNA - The Double Helix Game(Nobel Foundation) - Requires Flash Player - http://nobelprize.org/educational_games/medicine/dna_double_helix/
- Play the Genetic Code Game(Nobel Foundation) - Requires Flash Player - http://nobelprize.org/educational_games/medicine/gene-code/
- Gene-Environment Interaction Fact Sheet(Centers for Disease Control and Prevention) - http://www.cdc.gov/genomics/training/perspectives/factshts/geneenviro.htm
- ClinicalTrials.gov: Genes(National Institutes of Health) - http://clinicaltrials.gov/search/open/condition=%22Genes%22
- ClinicalTrials.gov: Gene Therapy(National Institutes of Health) - http://clinicaltrials.gov/search/open/intervention=%22gene+therapy%22
- 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
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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
- Article: Improving safety of gene therapy. - http://www.ncbi.nlm.nih.gov/sites/entrez?cmd=Retrieve&db=pubmed&dopt=AbstractPlus&list_uids=18690980&tool=MedlinePlus
- Article: Toward gene therapy of endometriosis: transductional and transcriptional targeting of... - http://www.ncbi.nlm.nih.gov/sites/entrez?cmd=Retrieve&db=pubmed&dopt=AbstractPlus&list_uids=18674655&tool=MedlinePlus
- Article: Optimizing exon skipping therapies for DMD. - http://www.ncbi.nlm.nih.gov/sites/entrez?cmd=Retrieve&db=pubmed&dopt=AbstractPlus&list_uids=18646569&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
- "-OMICS" Glossary: A Selection of Important Terms and Concepts Defined(Food and Drug Administration) - http://www.fda.gov/fdac/features/2005/605_glossary.html
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Talking Glossary of Genetic Terms(National Human Genome Research Institute) - Requires media player - http://www.genome.gov/glossary.cfm
Also available in Spanish http://www.genome.gov/sglossary.cfm
- Laboratory Directory-GeneReviews(University of Washington) - http://www.genetests.org/servlet/access?id=8888891&key=xW0l31Z2a4hTc&fcn=y&fw=XPF9&filename=/labsearch/searchdztest.html
- GeneTests(University of Washington) - http://www.genetests.org/
- National Human Genome Research Institute - http://www.genome.gov/
- Genomics & Health Weekly Update(Centers for Disease Control and Prevention) - http://www.cdc.gov/genomics/update/current.htm
- 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
- 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
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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
- 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/