Osteosarcoma, with poor survival after metastasis, is considered the most common primary
bone cancer in adolescents. Notwithstanding the efforts of researchers, its five-year survival …

Viral vectors have been used for a broad spectrum of gene therapy for both acute and
chronic diseases. In the context of cancer gene therapy, viral vectors expressing anti-tumor …

Background Gene therapy with elivaldogene autotemcel (eli-cel) consisting of autologous
CD34+ cells transduced with lentiviral vector containing ABCD1 complementary DNA (Lenti …

Despite the ups and downs in the field over three decades, the science of gene therapy has
continued to advance and provide enduring treatments for increasing number of diseases …

Clustered regularly interspersed short palindromic repeats (CRISPR)/CRISPR-associated
protein 9 (Cas9) technology has revolutionized the field of gene therapy as it has enabled …

Adenosine deaminase (ADA) deficiency leads to severe combined immunodeficiency
(SCID). Previous clinical trials showed that autologous CD34+ cell gene therapy (GT) …

Heterozygous mutations in CTLA-4 result in an inborn error of immunity with an autoimmune
and frequently severe clinical phenotype. Autologous T cell gene therapy may offer a cure …

Gene therapy has been widely investigated for the treatment of genetic, acquired, and
infectious diseases. Pioneering work utilized viral vectors; however, these are suspected of …

Inherited defects in the adenosine deaminase (ADA) gene typically cause severe combined
immunodeficiency. In addition to infections, ADA-deficient patients can present with …

Pompe disease is caused by deficiency of acid α-glucosidase (GAA), resulting in glycogen
accumulation in various tissues, including cardiac and skeletal muscles and the central …