Literature DB >> 34176774

InUtero Gene Therapy: Progress and Challenges.

Sourav K Bose1, Pallavi Menon1, William H Peranteau2.   

Abstract

In utero gene therapy has the potential to treat lethal and morbid perinatal diseases before birth. Small fetal size, a tolerogenic immune system, and dosing efficiency make the fetus a compelling patient. Numerous clinical, social, and institutional factors must be considered to achieve the promise of genetic treatment before birth.
Copyright © 2021 Elsevier Ltd. All rights reserved.

Entities:  

Keywords:  CRISPR; fetal; gene editing; gene therapy; in utero; lipid nanoparticle

Mesh：

Year:  2021        PMID： 34176774      PMCID： PMC8713252          DOI： 10.1016/j.molmed.2021.05.007

Source DB:  PubMed          Journal:  Trends Mol Med        ISSN： 1471-4914            Impact factor:   11.951


  11 in total

1.  Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems.

Authors:  C N Mattar; S N Waddington; A Biswas; N Johana; X W Ng; A S Fisk; N M Fisk; L G Tan; A A Rahim; S M K Buckley; M H Tan; J Lu; M Choolani; J K Y Chan
Journal:  Gene Ther       Date:  2012-01-26       Impact factor: 5.250

2.  Identification of preexisting adaptive immunity to Cas9 proteins in humans.

Authors:  Carsten T Charlesworth; Priyanka S Deshpande; Daniel P Dever; Joab Camarena; Viktor T Lemgart; M Kyle Cromer; Christopher A Vakulskas; Michael A Collingwood; Liyang Zhang; Nicole M Bode; Mark A Behlke; Beruh Dejene; Brandon Cieniewicz; Rosa Romano; Benjamin J Lesch; Natalia Gomez-Ospina; Sruthi Mantri; Mara Pavel-Dinu; Kenneth I Weinberg; Matthew H Porteus
Journal:  Nat Med       Date:  2019-01-28       Impact factor: 53.440

3.  Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system.

Authors:  Ahad A Rahim; Andrew M S Wong; Klemens Hoefer; Suzanne M K Buckley; Citra N Mattar; Seng H Cheng; Jerry K Y Chan; Jonathan D Cooper; Simon N Waddington
Journal:  FASEB J       Date:  2011-07-11       Impact factor: 5.191

4.  In utero gene editing for monogenic lung disease.

Authors:  Deepthi Alapati; William J Zacharias; Heather A Hartman; Avery C Rossidis; John D Stratigis; Nicholas J Ahn; Barbara Coons; Su Zhou; Hiaying Li; Kshitiz Singh; Jeremy Katzen; Yaniv Tomer; Alexandra C Chadwick; Kiran Musunuru; Michael F Beers; Edward E Morrisey; William H Peranteau
Journal:  Sci Transl Med       Date:  2019-04-17       Impact factor: 17.956

5.  Adeno-associated virus antibody profiles in newborns, children, and adolescents.

Authors:  Roberto Calcedo; Hiroki Morizono; Lili Wang; Robert McCarter; Jianping He; David Jones; Mark L Batshaw; James M Wilson
Journal:  Clin Vaccine Immunol       Date:  2011-07-20

6.  CD8(+) T-cell responses to adeno-associated virus capsid in humans.

Authors:  Federico Mingozzi; Marcela V Maus; Daniel J Hui; Denise E Sabatino; Samuel L Murphy; John E J Rasko; Margaret V Ragni; Catherine S Manno; Jurg Sommer; Haiyan Jiang; Glenn F Pierce; Hildegund C J Ertl; Katherine A High
Journal:  Nat Med       Date:  2007-03-18       Impact factor: 53.440

7.  Complications of intrauterine intravascular blood transfusion: lessons learned after 1678 procedures.

Authors:  C Zwiers; I T M Lindenburg; F J Klumper; M de Haas; D Oepkes; I L Van Kamp
Journal:  Ultrasound Obstet Gynecol       Date:  2017-08       Impact factor: 7.299

8.  In utero nanoparticle delivery for site-specific genome editing.

Authors:  Adele S Ricciardi; Raman Bahal; James S Farrelly; Elias Quijano; Anthony H Bianchi; Valerie L Luks; Rachael Putman; Francesc López-Giráldez; Süleyman Coşkun; Eric Song; Yanfeng Liu; Wei-Che Hsieh; Danith H Ly; David H Stitelman; Peter M Glazer; W Mark Saltzman
Journal:  Nat Commun       Date:  2018-06-26       Impact factor: 17.694

9.  Ionizable lipid nanoparticles for in utero mRNA delivery.

Authors:  Rachel S Riley; Meghana V Kashyap; Margaret M Billingsley; Brandon White; Mohamad-Gabriel Alameh; Sourav K Bose; Philip W Zoltick; Hiaying Li; Rui Zhang; Andrew Y Cheng; Drew Weissman; William H Peranteau; Michael J Mitchell
Journal:  Sci Adv       Date:  2021-01-13       Impact factor: 14.957

10.  In utero CRISPR-mediated therapeutic editing of metabolic genes.

Authors:  Avery C Rossidis; John D Stratigis; Alexandra C Chadwick; Heather A Hartman; Nicholas J Ahn; Haiying Li; Kshitiz Singh; Barbara E Coons; Li Li; Wenjian Lv; Philip W Zoltick; Deepthi Alapati; William Zacharias; Rajan Jain; Edward E Morrisey; Kiran Musunuru; William H Peranteau
Journal:  Nat Med       Date:  2018-10-08       Impact factor: 53.440
View more
  2 in total

Review 1.  Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies.

Authors:  Carsten W Lederer; Lola Koniali; Tina Buerki-Thurnherr; Panayiota L Papasavva; Stefania La Grutta; Amelia Licari; Frantisek Staud; Donato Bonifazi; Marina Kleanthous
Journal:  Pharmaceutics       Date:  2022-04-05       Impact factor: 6.525

Review 2.  Intrauterine Fetal Gene Therapy: Is That the Future and Is That Future Now?

Authors:  Nikhil Chowdary Peddi; Harshita Marasandra Ramesh; Sai Sravya Gude; Sai Sreeya Gude; Sravya Vuppalapati
Journal:  Cureus       Date:  2022-02-23
  2 in total

北京卡尤迪生物科技股份有限公司 © 2022-2023.