Gene, RNA, and ASO-based therapeutic approaches in Cystic Fibrosis

被引：13

作者：

Allaire, Normand E. ^{[1
]}

Griesenbach, Uta ^{[2
,3
]}

Kerem, Batsheva ^{[4
]}

Lueck, John D. ^{[5
]}

Stanleigh, Noemie ^{[4
]}

Oren, Yifat S. ^{[6
]}

机构：

[1] Cyst Fibrosis Fdn Lab, Lexington, MA USA

[2] Imperial Coll London, Natl Heart & Lung Inst, London, England

[3] UK Resp Gene Therapy Consortium, London, England

[4] Hebrew Univ Jerusalem, Life Sci Inst, Dept Genet, Jerusalem, Israel

[5] Univ Rochester, Dept Physiol & Pharmacol, Med Ctr, Rochester, NY USA

[6] Haddasah Karem, SpliSenseTherapeutics, Biohouse Labs, Jerusalem, Israel

来源：

JOURNAL OF CYSTIC FIBROSIS | 2023年 / 22卷

基金：

以色列科学基金会;

关键词：

Gene; RNA; ASO; Therapeutic; Cystic Fibrosis; Delivery; ANTISENSE OLIGONUCLEOTIDE; CFTR; VECTOR; SUPPRESSION; LENTIVIRUS; EPITHELIA; DELIVERY; MUTATION; W1282X; MOUSE;

D O I：

10.1016/j.jcf.2022.12.016

中图分类号：

R56 [呼吸系及胸部疾病];

学科分类号：

摘要：

Most people with Cystic Fibrosis (PwCF) harbor Cystic Fibrosis Transmembrane Conductance (CFTR) mutations that respond to highly effective CFTR modulators (HEM); however, a small fraction of non-responsive variants will require alternative approaches for treatment. Furthermore, the long-term goal to develop a cure for CF will require novel therapeutic strategies. Nucleic acid-based approaches offer the potential to address all CF-causing mutations and possibly a cure for all PwCF. In this minireview, we discuss current knowledge, recent progress, and critical questions surrounding the topic of Gene-, RNA-, and ASO-based therapies for the treatment of Cystic Fibrosis (CF). (C) 2023 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.

引用

页码：S39 / S44

页数：6

共 46 条

[1]

4dmoleculartherapeutics, US

[2] Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis [J].

Alton, Eric W. F. W. ;

Beekman, Jeffery M. ;

Boyd, A. Christopher ;

Brand, June ;

Carlon, Marianne S. ;

Connolly, Mary M. ;

Chan, Mario ;

Conlon, Sinead ;

Davidson, Heather E. ;

Davies, Jane C. ;

Davies, Lee A. ;

Dekkers, Johanna F. ;

Doherty, Ann ;

Gea-Sorli, Sabrina ;

Gill, Deborah R. ;

Griesenbach, Uta ;

Hasegawa, Mamoru ;

Higgins, Tracy E. ;

Hironaka, Takashi ;

Hyndman, Laura ;

McLachlan, Gerry ;

Inoue, Makoto ;

Hyde, Stephen C. ;

Innes, J. Alastair ;

Maher, Toby M. ;

Moran, Caroline ;

Meng, Cuixiang ;

Paul-Smith, Michael C. ;

Pringle, Ian A. ;

Pytel, Kamila M. ;

Rodriguez-Martinez, Andrea ;

Schmidt, Alexander C. ;

Stevenson, Barbara J. ;

Sumner-Jones, Stephanie G. ;

Toshner, Richard ;

Tsugumine, Shu ;

Wasowicz, Marguerite W. ;

Zhu, Jie .

THORAX, 2017, 72 (02) :137-147

[3] Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial [J].

Alton, Eric W. F. W. ;

Armstrong, David K. ;

Ashby, Deborah ;

Bayfield, Katie J. ;

Bilton, Diana ;

Bloomfield, Emily V. ;

Boyd, A. Christopher ;

Brand, June ;

Buchan, Ruaridh ;

Calcedo, Roberto ;

Carvelli, Paula ;

Chan, Mario ;

Cheng, Seng H. ;

Collie, D. David S. ;

Cunningham, Steve ;

Davidson, Heather E. ;

Davies, Gwyneth ;

Davies, Jane C. ;

Davies, Lee A. ;

Dewar, Maria H. ;

Doherty, Ann ;

Donovan, Jackie ;

Dwyer, Natalie S. ;

Elgmati, Hala I. ;

Featherstone, Rosanna F. ;

Gavino, Jemyr ;

Gea-Sorli, Sabrina ;

Geddes, Duncan M. ;

Gibson, James S. R. ;

Gill, Deborah R. ;

Greening, Andrew P. ;

Griesenbach, Uta ;

Hansell, David M. ;

Harman, Katharine ;

Higgins, Tracy E. ;

Hodges, Samantha L. ;

Hyde, Stephen C. ;

Hyndman, Laura ;

Innes, J. Alastair ;

Jacob, Joseph ;

Jones, Nancy ;

Keogh, Brian F. ;

Limberis, Maria P. ;

Lloyd-Evans, Paul ;

Maclean, Alan W. ;

Manvell, Michelle C. ;

McCormick, Dominique ;

McGovern, Michael ;

McLachlan, Gerry ;

Meng, Cuixiang .

LANCET RESPIRATORY MEDICINE, 2015, 3 (09) :684-691

[4]

[Anonymous], ABOUT US

[5] Potential of helper-dependent Adenoviral vectors in CRISPR-cas9-mediated lung gene therapy [J].

Bandara, Ranmal Avinash ;

Chen, Ziyan Rachel ;

Hu, Jim .

CELL AND BIOSCIENCE, 2021, 11 (01)

[6]

boehringer, about us

[7] Transducing Airway Basal Cells with a Helper-Dependent Adenoviral Vector for Lung Gene Therapy [J].

Cao, Huibi ;

Ouyang, Hong ;

Grasemann, Hartmut ;

Bartlett, Claire ;

Du, Kai ;

Duan, Rongqi ;

Shi, Fushan ;

Estrada, Marvin ;

Seigel, Kyle E. ;

Coates, Allan L. ;

Yeger, Herman ;

Bear, Christine E. ;

Gonska, Tanja ;

Moraes, Theo J. ;

Hu, Jim .

HUMAN GENE THERAPY, 2018, 29 (06) :643-652

[8] Novel Lung Tropic Adeno-Associated Virus Capsids for Therapeutic Gene Delivery [J].

Carneiro, Ana ;

Lee, Hyuncheol ;

Lin, Li ;

van Haasteren, Joost ;

Schaffer, David V. .

HUMAN GENE THERAPY, 2020, 31 (17-18) :996-1009

[9]

cff.org, US

[10]

Clinicaltrials, Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis (RESTORE-CF)

← 1 2 3 4 5 →