Title:Destination Penis? Gene Therapy as a Possible Treatment for Erectile Dysfunction
Volume: 18
Issue: 4
关键词:
勃起功能障碍，基因治疗，病毒载体，车辆系统，编辑技术，心血管疾病。
摘要: Erectile Dysfunction (ED) is a common health condition occuring in roughly 50% of aging
males (40-70 years old). Recent attention has related gene therapy to ED, and now there is an interest
to further implement gene therapy concepts to ED treatment. This review is an attempt to analyze key
challenges and emphasize primary areas, including mostly preclinical and a few clinical trials, cellular
target(s), and different viral vectors/nanoparticles for gene delivery in ED. While overexpression of
target genes can be silenced by RNA interference (RNAi), down-regulation of these mechanisms has
been implicated in ED. Although many patients with ED demonstrate efficacy with phosphodiesterase
type 5 inhibitors, this therapy is insufficient in approximately 30-40% of patients. Although several
preclinical studies for ED treatment provided promising results, gene therapy has not shown success in
clinical practice, due in part to technical limitations of gene therapy to address ED pathogenesis. Developments
in small RNA, such as small interfering RNA (siRNA) may lead to significant benefit in
the management of ED. Also, siRNA delivery into the corpus cavernosum seems a challenging issue
and awaits further development. Several safety concerns of gene therapy, gene acquisition, preparation,
and delivery are necessary to continue investigation before any widespread application is used in
ED treatment.
