Bolstad Anne Isine, Jonsson Roland
Department of Oral Sciences-Periodontics, Faculty of Dentistry, University of Bergen, Aarstadveien 17, N-5009 Bergen, Norway.
Expert Opin Biol Ther. 2005 Jun;5(6):763-72. doi: 10.1517/14712598.5.6.763.
Sjögren's syndrome (SS) is a complex autoimmune disorder, characterised by mononuclear cell infiltration of exocrine glands, principally the lacrimal and salivary glands. Both cellular, in the form of autoreactive immune cells, and humoral factors, such as autoantibodies, contribute to the expression of the disease. SS can also occur as a systemic disease affecting several organs, and approximately 5% of the patients develop malignant lymphoproliferation. Today SS is considered uncurable. The treatment available is only palliative, and the treatment goals are to manage symptoms and prevent or limit tissue damage. This may involve both local and systemic measures. However, the existing systemic treatment of chronic inflammatory autoimmune diseases has several limitations and unwanted side effects. In recent years the possibility to treat diseases with gene therapy has gained interest and has become a subject of investigation. Given the multitude of factors contributing to the pathogenesis of SS, gene therapy is a major challenge, but may elicit great benefits if successful. Keeping this in mind, the possibility for gene therapeutics in SS in general and potential targets for gene therapy are discussed.
干燥综合征(SS)是一种复杂的自身免疫性疾病,其特征是外分泌腺,主要是泪腺和唾液腺出现单核细胞浸润。自身反应性免疫细胞形式的细胞因素和自身抗体等体液因素均促成该疾病的表现。SS也可作为一种影响多个器官的全身性疾病出现,约5%的患者会发生恶性淋巴增殖。如今,SS被认为无法治愈。现有的治疗仅为姑息性治疗,治疗目标是控制症状并预防或限制组织损伤。这可能涉及局部和全身措施。然而,现有的慢性炎症性自身免疫性疾病的全身治疗存在若干局限性和不良副作用。近年来,用基因疗法治疗疾病的可能性引起了关注,并已成为一个研究课题。鉴于导致SS发病机制的因素众多,基因疗法是一项重大挑战,但如果成功可能会带来巨大益处。考虑到这一点,本文讨论了SS中基因治疗的总体可能性以及基因治疗的潜在靶点。