After setbacks related to serious adverse events 20 years ago, gene therapy is now coming
back to the central stage worldwide. In the past few years, gene therapy has shown …

A novel approach in gene therapy was introduced 20 years ago since artificial non-
integrative chromosome-based vectors containing gene loci size inserts were engineered …

Nucleic acid-based therapeutics are currently developed at large scale for prevention and
management of cardiovascular diseases (CVDs), since:(i) genetic studies have highlighted …

Hereditary antithrombin deficiency is caused by SERPINC1 gene mutations and
predisposes to recurrent venous thromboembolism that can be life-threatening. Therefore …

Hemophilia A (HA), a common bleeding disorder caused by a deficiency of coagulation
factor VIII (FVIII), has long been considered an attractive target for gene therapy studies …

Clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9
(Cas9) is one of the most versatile and efficient gene editing technologies, which is derived …

Juvenile myelomonocytic leukemia (JMML) is a malignant myeloproliferative disorder
arising in infants and young children. The origin of this neoplasm is attributed to an early …

Background Treatment of hemophiliacs with inhibitors remains challenging, and new
treatments are in urgent need. Coagulation factor X plays a critical role in the downstream of …

Hemophilia A (HA) is a hereditary hemorrhagic disease caused by a deficiency of
coagulation factor VIII (FVIII) in blood plasma. Patients with HA usually suffer from …

El desarrollo de tecnologías para la edición del genoma ha abierto la posibilidad de apuntar
directamente y modificar secuencias genómicas en casi todo tipo de células eucariotas. La …