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Gene Therapy
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Zinc Finger Nucleases (ZFNs)
ZFNs are engineered DNA-binding proteins that facilitate targeted editing of the genome by creating double-strand breaks in DNA at user-specified locations. They are used for gene therapy in HIV, by disabling CCR5 receptor gene.
Lentiviral Vectors
Lentiviral vectors are delivery systems derived from the Lentivirus genus, capable of integrating new genes into the host genome. They are used in gene therapy for inherited and acquired diseases.
Ex Vivo Gene Therapy
Ex vivo gene therapy involves modifying the genes in cells that have been removed from the patient, followed by re-implanting them. This technique is applied in treating blood disorders, such as thalassemia.
Induced Pluripotent Stem Cells (iPSCs)
iPSCs are a type of pluripotent stem cell generated directly from adult cells. They are used in gene therapy to replace diseased tissues with genetically corrected healthy ones.
In Vivo Gene Therapy
In vivo gene therapy delivers genes directly into cells within the patient's body. It has potential applications in treating a wide range of diseases, including cystic fibrosis and certain types of cancer.
Somatic Gene Therapy
Somatic gene therapy involves inserting therapeutic genes into the somatic (body) cells of a patient. This does not affect offspring and is more ethically accepted. It is used for treating diseases like cancer.
Adeno-Associated Virus (AAV) Vectors
AAV vectors are small viruses that infect humans but are not known to cause disease. They are used as delivery vehicles for gene therapy, introducing new genes to treat conditions like hemophilia and muscular dystrophy.
Homologous Recombination
Homologous recombination is a mechanism for introducing genetic changes by exchange of DNA sequences between similar or identical molecules of DNA. It is harnessed in gene therapy for precise gene editing.
Germline Gene Therapy
Germline gene therapy involves modifying the genes in sperm, eggs, or embryos. This affects all subsequent generations and carries ethical considerations. It has potential for eradicating genetic diseases before birth.
Gene Silencing
Gene silencing is the regulation of gene expression to prevent the transcription and translation of a certain gene. It can be used to reduce the production of harmful proteins in diseases like Huntington's.
Non-Homologous End Joining (NHEJ)
NHEJ is a pathway that repairs double-strand breaks in DNA by directly joining the broken ends. It is less precise than homologous recombination and can be used in gene disruption as a form of therapy.
CRISPR-Cas9
CRISPR-Cas9 is a genome editing tool that allows for precise, directed changes to genomic DNA. It has potential applications in treating genetic disorders, by correcting the genetic mutations.
TAL Effector Nucleases (TALENs)
TALENs are restriction enzymes that can be engineered to cut specific sequences of DNA. They have potential applications in correcting single-gene disorders like sickle cell anemia.
RNA Interference (RNAi)
RNA interference is a biological process in which RNA molecules inhibit gene expression. Its applications in gene therapy include targeting and silencing deleterious genes involved in disease.
Antisense Oligonucleotides
Antisense oligonucleotides are short, synthetic strands of DNA or RNA that can bind to RNA and alter gene expression. They are used to inhibit disease-causing proteins or to modify splicing patterns.
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