In the context of gene therapy, the term “compromised genomics” generally refers to situations where the underlying genetic information or genomic characteristics of an individual pose challenges or limitations for the successful implementation of gene therapy.
Gene therapy involves the delivery of therapeutic genes into a patient’s cells to treat or correct a genetic disorder. However, the success of gene therapy can be influenced by various factors related to the patient’s genomic profile. “Compromised genomics” may refer to:
Genetic Mutations:
Genetic mutations: If a patient has specific genetic mutations that directly or indirectly affect the functioning or availability of the targeted gene or its related pathways, it can compromise the effectiveness of gene therapy. These mutations may interfere with the expression of the therapeutic gene or impede the desired correction or restoration of the genetic defect.
Genomic Instability:
Genomic instability: Some individuals may have an inherently unstable or mutated genome, making it more challenging to achieve long-term stability and expression of the introduced therapeutic genes. Genomic instability can include factors such as increased chromosomal rearrangements, DNA replication errors, or defective DNA repair mechanisms.
Immune Responses:
Immune responses: Gene therapy approaches often involve the use of viral vectors or other delivery systems to introduce therapeutic genes into the patient’s cells. In some cases, a patient’s immune system may mount an immune response against the vector or the transgene, leading to reduced efficacy or even adverse reactions. This immune response can be influenced by the patient’s individual genetic background, including their human leukocyte antigen (HLA) profile, which is involved in immune recognition and response.
Understanding Compromised Genomics:Addressing compromised genomics in gene therapy requires careful consideration of the specific genetic and genomic characteristics of each patient. Personalized approaches, such as optimizing the choice of viral vectors, modifying the delivery methods, or developing strategies to mitigate immune responses, may be necessary to overcome these challenges and enhance the effectiveness of gene therapy in individuals with compromised genomics.
