Treating genetic disease

From metabolic care and enzyme replacement to gene therapy and editing.

6 pages

• Treatment of genetic disease: from diet to gene therapyGenetic diseases are treated at every level, from the diet and the missing product to the…
• Newborn screeningA few drops of heel blood detect treatable diseases before they cause harm, such as congenital…
• Lysosomal storage diseasesWhen a lysosomal enzyme is missing, the molecule it should break down stays inside, the lysosome…
• Gene therapyGene therapy treats a disease by adding, silencing or correcting a gene inside the patient's…
• CRISPR-Cas9 and genome editingCRISPR-Cas9 is RNA-guided 'molecular scissors' that cut DNA at an exact spot to switch off…
• MicroRNA, RNA interference and antisense drugsShort RNAs can switch off a gene after it has been copied into mRNA; cells use them naturally…

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This section is an index: it gathers pages from Biochemistry, Pathology, Embryology and other sections without repeating them. Educational content; it does not replace a geneticist's assessment or genetic counselling.

Representative model, for educational purposes only.