Hereditary diseases arise from germline mutations or large chromosomal aberrations that perturbs the normal cellular processes. These mutations and chromosomal abnormalities disrupt the expression of a single or a group of genes that encode for effector proteins performing critical cellular functions by inhibiting protein synthesis or production of dysfunctional copies of the protein. Based on their inheritance pattern, they can be grouped as autosomal dominant, autosomal recessive, X-linked, or mitochondrial. Traditional therapeutic approaches have primarily focused on managing their clinical symptoms rather than focusing on the underlying genetic anomalies. In this regard, gene therapy has transformed the treatment regimens for these disorders, by offering strategies such as correcting the underlying faulty gene, introduction of a functional copy of the gene or modulation of host factors to minimize the effects of the mutation. These approaches go hand to hand with incremental development in gene delivery and editing tools such as vector-mediated gene delivery, base-editing, CRISPR/Cas9, antisense oligonucleotides, and RNA interference. Most of the gene editing therapies are increasingly being approved clinically, which marks the therapeutic potential of these innovative methods. Additionally, current clinical trials for immunodeficiencies, hemoglobinopathies, cystic fibrosis, and Huntington’s disease, highlight the potential for long-term therapeutic benefit through targeted gene function restoration. However, despite significant progress, challenges persist regarding efficiency of gene delivery, undesirable host immune response against vectors, and long-term safety, which underscores the need for deeper research combined with necessary ethical oversight.