Literature DB >> 9326649

A factor IX-deficient mouse model for hemophilia B gene therapy.

L Wang1, M Zoppè, T M Hackeng, J H Griffin, K F Lee, I M Verma.   

Abstract

We have generated a mouse where the clotting factor IX (FIX) gene has been disrupted by homologous recombination. The FIX nullizygous (-/-) mouse was devoid of factor IX antigen in plasma. Consistent with the bleeding disorder, the factor IX coagulant activities for wild-type (+/+), heterozygous (+/-), and homozygous (-/-) mice were 92%, 53%, and <5%, respectively, in activated partial thromboplastin time assays. Plasma factor IX activity in the deficient mice (-/-) was restored by introducing wild-type murine FIX gene via adenoviral vectors. Thus, these factor IX-deficient mice provide a useful animal model for gene therapy studies of hemophilia B.

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Year:  1997        PMID： 9326649      PMCID： PMC23538          DOI： 10.1073/pnas.94.21.11563

Source DB:  PubMed          Journal:  Proc Natl Acad Sci U S A        ISSN： 0027-8424            Impact factor:   11.205


  22 in total

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Journal:  Br Med J       Date:  1952-12-27

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Journal:  Gene       Date:  1990-02-14       Impact factor: 3.688

Review 3.  Structure and function of factor IX: defects in haemophilia B.

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Journal:  Thromb Haemost       Date:  1995-07       Impact factor: 5.249

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Journal:  Cell       Date:  1991-06-28       Impact factor: 41.582

6.  Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression.

Authors:  Y Dai; E M Schwarz; D Gu; W W Zhang; N Sarvetnick; I M Verma
Journal:  Proc Natl Acad Sci U S A       Date:  1995-02-28       Impact factor: 11.205

7.  Genetically modified skin fibroblasts persist long after transplantation but gradually inactivate introduced genes.

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Journal:  Proc Natl Acad Sci U S A       Date:  1991-02-15       Impact factor: 11.205

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Authors:  D St Louis; I M Verma
Journal:  Proc Natl Acad Sci U S A       Date:  1988-05       Impact factor: 11.205

10.  MHC class I-restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with E1-deleted recombinant adenoviruses.

Authors:  Y Yang; H C Ertl; J M Wilson
Journal:  Immunity       Date:  1994-08       Impact factor: 31.745
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  54 in total

Review 1.  Gene therapy for the hemophilias.

Authors:  M A Kay; K High
Journal:  Proc Natl Acad Sci U S A       Date:  1999-08-31       Impact factor: 11.205

Review 2.  Adeno-associated virus vectors and hematology.

Authors:  D W Russell; M A Kay
Journal:  Blood       Date:  1999-08-01       Impact factor: 22.113

Review 3.  Adeno-associated virus-mediated gene transfer for hemophilia B.

Authors:  Katherine A High
Journal:  Int J Hematol       Date:  2002-11       Impact factor: 2.490

Review 4.  Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.

Authors:  Federico Mingozzi; Katherine A High
Journal:  Nat Rev Genet       Date:  2011-05       Impact factor: 53.242

5.  Therapeutic potential of spheroids of stem cells from human exfoliated deciduous teeth for chronic liver fibrosis and hemophilia A.

Authors:  Yoshiaki Takahashi; Ratih Yuniartha; Takayoshi Yamaza; Soichiro Sonoda; Haruyoshi Yamaza; Kosuke Kirino; Koichiro Yoshimaru; Toshiharu Matsuura; Tomoaki Taguchi
Journal:  Pediatr Surg Int       Date:  2019-09-24       Impact factor: 1.827

6.  Muscle-directed gene therapy for hemophilia B with more efficient and less immunogenic AAV vectors.

Authors:  L Wang; J-P Louboutin; P Bell; J A Greig; Y Li; D Wu; J M Wilson
Journal:  J Thromb Haemost       Date:  2011-10       Impact factor: 5.824

7.  Recombinant, replication-defective adenovirus gene transfer vectors induce cell cycle dysregulation and inappropriate expression of cyclin proteins.

Authors:  R P Wersto; E R Rosenthal; P K Seth; N T Eissa; R E Donahue
Journal:  J Virol       Date:  1998-12       Impact factor: 5.103

8.  Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy.

Authors:  Nisha Nair; Melvin Y Rincon; Hanneke Evens; Shilpita Sarcar; Sumitava Dastidar; Emira Samara-Kuko; Omid Ghandeharian; Hiu Man Viecelli; Beat Thöny; Pieter De Bleser; Thierry VandenDriessche; Marinee K Chuah
Journal:  Blood       Date:  2014-03-17       Impact factor: 22.113

9.  Tissue factor is required for uterine hemostasis and maintenance of the placental labyrinth during gestation.

Authors:  J Erlich; G C Parry; C Fearns; M Muller; P Carmeliet; T Luther; N Mackman
Journal:  Proc Natl Acad Sci U S A       Date:  1999-07-06       Impact factor: 11.205

10.  A mouse model of severe von Willebrand disease: defects in hemostasis and thrombosis.

Authors:  C Denis; N Methia; P S Frenette; H Rayburn; M Ullman-Culleré; R O Hynes; D D Wagner
Journal:  Proc Natl Acad Sci U S A       Date:  1998-08-04       Impact factor: 11.205
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