Gene Therapy: A New Hope in Sickle Cell Disease Treatment

被引：0

作者：

Christy, Margaret ^{[1
,2
]}

Fisher, Beth ^{[1
,3
,4
,5
]}

机构：

[1] Clemson Univ, Clemson, SC USA

[2] Prisma Hlth Upstate, 605 Grove Rd, Greenville, SC 29605 USA

[3] Wellstar MCG Hlth Childrens Hosp Georgia, Pediat Palliat Care Program, Augusta, GA USA

[4] Wellstar MCG Hlth Childrens Hosp Georgia, Childhood Canc Survivorship Program, Augusta, GA USA

[5] Childrens Healthcare Atlanta, Atlanta, GA USA

来源：

JOURNAL OF PEDIATRIC HEALTH CARE | 2025年 / 39卷 / 01期

关键词：

Gene therapy; sickle cell disease; adolescence; CRISPR; lentiviral vector; MANAGEMENT; STATES;

D O I：

10.1016/j.pedhc.2024.07.007

中图分类号：

R19 [保健组织与事业（卫生事业管理）];

学科分类号：

摘要：

Sickle cell disease (SCD) is a lifelong disease requiring expensive treatment for management and limited curative options until the last few years. Gene therapy has emerged as a curative option for SCD, with two approved therapies available to SCD patients aged >= 12 years. Consideration must be considered regarding the ethics, efficacy, management requirements, education, and counseling needs of patients and their parents. Current and future practices will need to advocate for improved access and affordability of this specialized care and address the unknown and less defined areas of gene therapy pertaining to SCD through research.

引用

页码：122 / 129

页数：8

共 24 条

[1]

American Society of Hematology, 2024, ASH supports CMMI's cell & gene therapy access model for sickle cell disease

[2]

[Anonymous], NATL HEART LUNG BLOO

[3]

[Anonymous], Tugwell, Rexford Guy. Interview by Charles O. Jackson. June 7, 1968, U.S. Food and Drug Administration Oral History, 14-15. https://www.fda.gov/media/81126/download/.

[4]

[Anonymous], Centers for Disease Control and Prevention. (2021). https://www.cdc.gov/index.html

[5]

Centers for Disease Control and Prevention, 2023, What is sickle cell disease?

[6]

Centers for Medicaid and Medicare Innovation, 2024, Cell and Gene Therapy Access Model

[7] Exagamglogene Autotemcel for Severe Sickle Cell Disease [J].

Frangoul, Haydar ;

Locatelli, Franco ;

Sharma, Akshay ;

Bhatia, Monica ;

Mapara, Markus ;

Molinari, Lyndsay ;

Wall, Donna ;

Liem, Robert I. ;

Telfer, Paul ;

Shah, Ami J. ;

Cavazzana, Marina ;

Corbacioglu, Selim ;

Rondelli, Damiano ;

Meisel, Roland ;

Dedeken, Laurence ;

Lobitz, Stephan ;

de Montalembert, Mariane ;

Steinberg, Martin H. ;

Walters, Mark C. ;

Bower, Laura ;

Imren, Suzan ;

Simard, Christopher ;

Xuan, Fengjuan ;

Zhou, Weiyu ;

Morrow, Phuong Khanh ;

Hobbs, William ;

Grupp, Stephan .

BLOOD, 2023, 142

[8]

GBD, 2021, Lancet Haematol, V10, P585, DOI [10.1016/S2352-3026(23)00118-7, DOI 10.1016/S2352-3026(23)00118-7]

[9] Lifetime medical costs attributable to sickle cell disease among nonelderly individuals with commercial insurance [J].

Johnson, Kate M. ;

Jiao, Boshen ;

Ramsey, Scott D. ;

Bender, M. A. ;

Devine, Beth ;

Basu, Anirban .

BLOOD ADVANCES, 2023, 7 (03) :365-374

[10] Lovo-cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB-206 study [J].

Kanter, Julie ;

Thompson, Alexis A. ;

Pierciey, Francis J., Jr. ;

Hsieh, Matthew ;

Uchida, Naoya ;

Leboulch, Philippe ;

Schmidt, Manfred ;

Bonner, Melissa ;

Guo, Ruiting ;

Miller, Alex ;

Ribeil, Jean-Antoine ;

Davidson, David ;

Asmal, Mohammed ;

Walters, Mark C. ;

Tisdale, John F. .

AMERICAN JOURNAL OF HEMATOLOGY, 2023, 98 (01) :11-22

← 1 2 3 →