Final answer:
Gene therapy, including the use of antisense oligonucleotides, is a treatment strategy for β-thalassemia that involves correcting the mutant splice site or the introduction of a functional gene into the patient's genome to compensate for the defective one.
Step-by-step explanation:
Correction of a mutant splice site with an antisense oligonucleotide can be used to treat β-thalassemia. Gene therapy is an experimental method of curing genetic disorders by inserting normal genes into cells with mutant genes. This technique involves the introduction of a nonmutated functional gene to compensate for the defective one, or it can involve correcting the genetic mutation at the original site on the genome.
In the case of β-thalassemia, which can be caused by various mutations including those affecting the beta-globin gene's promoter region or splice sites, such therapies can be crucial in enhancing the synthesis rate of the affected protein. Moreover, efficient detection methodologies allow for identification of mutations at multiple sites within a single gene, facilitating targeted gene therapy approaches.