Literature DB >> 30837116

In Utero Gene Therapy Consensus Statement from the IFeTIS.

Graça Almeida-Porada1, Simon N Waddington2, Jerry K Y Chan3, William H Peranteau4, Tippi MacKenzie5, Christopher D Porada6.   

Abstract

Mesh：

Year:  2019        PMID： 30837116      PMCID： PMC6453510          DOI： 10.1016/j.ymthe.2019.02.015

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


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  10 in total

1.  Prenatal gene tranfer: scientific, medical, and ethical issues: a report of the Recombinant DNA Advisory Committee.

Authors: 
Journal:  Hum Gene Ther       Date:  2000-05-01       Impact factor: 5.695

2.  Inherited metabolic disorders: prenatal diagnosis of lysosomal storage disorders.

Authors:  Jyotsna Verma; Divya C Thomas; Sandeepika Sharma; Geetu Jhingan; Renu Saxena; Sudha Kohli; Ratna D Puri; Sunita Bijarnia; Ishwar C Verma
Journal:  Prenat Diagn       Date:  2015-09-03       Impact factor: 3.050

3.  Two Decades of Molecular Therapy: The Journey Continues.

Authors:  Robert M Frederickson; Seppo Ylä-Herttuala
Journal:  Mol Ther       Date:  2018-12-11       Impact factor: 11.454

Review 4.  Fetal stem cell and gene therapy.

Authors:  Russell Witt; Tippi C MacKenzie; William H Peranteau
Journal:  Semin Fetal Neonatal Med       Date:  2017-06-12       Impact factor: 3.926

5.  The Next Phase of Human Gene-Therapy Oversight.

Authors:  Francis S Collins; Scott Gottlieb
Journal:  N Engl J Med       Date:  2018-08-15       Impact factor: 91.245

6.  In utero gene therapy: transfer and long-term expression of the bacterial neo(r) gene in sheep after direct injection of retroviral vectors into preimmune fetuses.

Authors:  C D Porada; N Tran; M Eglitis; R C Moen; L Troutman; A W Flake; Y Zhao; W F Anderson; E D Zanjani
Journal:  Hum Gene Ther       Date:  1998-07-20       Impact factor: 5.695

7.  Prenatal Correction of X-Linked Hypohidrotic Ectodermal Dysplasia.

Authors:  Holm Schneider; Florian Faschingbauer; Sonia Schuepbach-Mallepell; Iris Körber; Sigrun Wohlfart; Angela Dick; Mandy Wahlbuhl; Christine Kowalczyk-Quintas; Michele Vigolo; Neil Kirby; Corinna Tannert; Oliver Rompel; Wolfgang Rascher; Matthias W Beckmann; Pascal Schneider
Journal:  N Engl J Med       Date:  2018-04-26       Impact factor: 91.245

8.  Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector-mediated intrauterine gene transfer in early-gestation fetal macaques.

Authors:  Jerry K Y Chan; Irene Gil-Farina; Nuryanti Johana; Cecilia Rosales; Yi Wan Tan; Jessika Ceiler; Jenny Mcintosh; Bryan Ogden; Simon N Waddington; Manfred Schmidt; Arijit Biswas; Mahesh Choolani; Amit C Nathwani; Citra N Z Mattar
Journal:  FASEB J       Date:  2018-12-05       Impact factor: 5.191

Review 9.  In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application.

Authors:  Graça Almeida-Porada; Anthony Atala; Christopher D Porada
Journal:  Mol Ther Methods Clin Dev       Date:  2016-03-30       Impact factor: 6.698

10.  Fetal gene therapy for neurodegenerative disease of infants.

Authors:  Giulia Massaro; Citra N Z Mattar; Andrew M S Wong; Ernestas Sirka; Suzanne M K Buckley; Bronwen R Herbert; Stefan Karlsson; Dany P Perocheau; Derek Burke; Simon Heales; Angela Richard-Londt; Sebastian Brandner; Mylene Huebecker; David A Priestman; Frances M Platt; Kevin Mills; Arijit Biswas; Jonathan D Cooper; Jerry K Y Chan; Seng H Cheng; Simon N Waddington; Ahad A Rahim
Journal:  Nat Med       Date:  2018-07-16       Impact factor: 53.440
  10 in total
  13 in total

1.  Molecular Diagnostics and In Utero Therapeutics for Orofacial Clefts.

Authors:  J D Oliver; E C Turner; L R Halpern; S Jia; P Schneider; R N D'Souza
Journal:  J Dent Res       Date:  2020-07-01       Impact factor: 6.116

2.  Investigating Optimal Autologous Cellular Platforms for Prenatal or Perinatal Factor VIII Delivery to Treat Hemophilia A.

Authors:  Christopher Stem; Christopher Rodman; Ritu M Ramamurthy; Sunil George; Diane Meares; Andrew Farland; Anthony Atala; Christopher B Doering; H Trent Spencer; Christopher D Porada; Graça Almeida-Porada
Journal:  Front Cell Dev Biol       Date:  2021-08-10

Review 3.  Spinal muscular atrophy.

Authors:  Eugenio Mercuri; Charlotte J Sumner; Francesco Muntoni; Basil T Darras; Richard S Finkel
Journal:  Nat Rev Dis Primers       Date:  2022-08-04       Impact factor: 65.038

Review 4.  Delivery technologies for in utero gene therapy.

Authors:  Rohan Palanki; William H Peranteau; Michael J Mitchell
Journal:  Adv Drug Deliv Rev       Date:  2020-11-09       Impact factor: 15.470

5.  Mechanistic Insights into Factor VIII Immune Tolerance Induction via Prenatal Cell Therapy in Hemophilia A.

Authors:  Martin Rodriguez; Christopher D Porada; Graҫa Almeida-Porada
Journal:  Curr Stem Cell Rep       Date:  2019-11-20

6.  Nanoparticles for delivery of agents to fetal lungs.

Authors:  Sarah J Ullrich; Mollie Freedman-Weiss; Samantha Ahle; Hanna K Mandl; Alexandra S Piotrowski-Daspit; Katherine Roberts; Nicholas Yung; Nathan Maassel; Tory Bauer-Pisani; Adele S Ricciardi; Marie E Egan; Peter M Glazer; W Mark Saltzman; David H Stitelman
Journal:  Acta Biomater       Date:  2021-01-21       Impact factor: 8.947

7.  Current and emerging pharmacotherapy for Gaucher disease in pediatric populations.

Authors:  Richard Sam; Emory Ryan; Emily Daykin; Ellen Sidransky
Journal:  Expert Opin Pharmacother       Date:  2021-03-25       Impact factor: 4.103

Review 8.  The Future of In Utero Gene Therapy.

Authors:  William H Peranteau; Alan W Flake
Journal:  Mol Diagn Ther       Date:  2020-04       Impact factor: 4.476

9.  Defining the Optimal FVIII Transgene for Placental Cell-Based Gene Therapy to Treat Hemophilia A.

Authors:  Nadia El-Akabawy; Martin Rodriguez; Ritu Ramamurthy; Andrew Rabah; Brady Trevisan; Alshaimaa Morsi; Sunil George; Jordan Shields; Diane Meares; Andrew Farland; Anthony Atala; Christopher B Doering; H Trent Spencer; Christopher D Porada; Graça Almeida-Porada
Journal:  Mol Ther Methods Clin Dev       Date:  2020-03-14       Impact factor: 6.698

10.  Prenatal genetic diagnosis: Fetal therapy as a possible solution to a positive test.

Authors:  Aysha Karim Kiani; Stefano Paolacci; Pietro Scanzano; Sandro Michelini; Natale Capodicasa; Leonardo D'Agruma; Angelantonio Notarangelo; Gerolamo Tonini; Daniela Piccinelli; Kalantary Rad Farshid; Paolo Petralia; Ezio Fulcheri; Francesca Buffelli; Pietro Chiurazzi; Corrado Terranova; Francesco Plotti; Roberto Angioli; Marco Castori; Ondrej Pös; Tomas Szemes; Matteo Bertelli
Journal:  Acta Biomed       Date:  2020-11-09
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