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Bernd Hauck

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Articles 16
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Recent Articles
1.
Bevaart L, Aalbers C, Vierboom M, Broekstra N, Kondova I, Breedveld E, et al.
Hum Gene Ther Clin Dev . 2015 Jun; 26(2):103-12. PMID: 26086763
Preclinical studies to assess biodistribution, safety, and initial efficacy of ART-I02, an adeno-associated type 5 (rAAV5) vector expressing human interferon β (hIFN-β), were performed in a total of 24 rhesus...
2.
Dong B, Duan X, Chow H, Chen L, Lu H, Wu W, et al.
PLoS One . 2014 Feb; 9(2):e86453. PMID: 24498275
Recombinant adeno-associated vectors (rAAV) are commonly purified by either chromatography or equilibrium CsCl gradient. Nevertheless, even after purification various cellular proteins often associate with rAAV vector capsids. Such co-purifying cellular...
3.
Grupp S, Kalos M, Barrett D, Aplenc R, Porter D, Rheingold S, et al.
N Engl J Med . 2013 Mar; 368(16):1509-1518. PMID: 23527958
Chimeric antigen receptor-modified T cells with specificity for CD19 have shown promise in the treatment of chronic lymphocytic leukemia (CLL). It remains to be established whether chimeric antigen receptor T...
4.
Suhy D, Kao S, Mao T, Whiteley L, Denise H, Souberbielle B, et al.
Mol Ther . 2012 Jun; 20(9):1737-1749. PMID: 22735378
The hepatitis C virus (HCV) chronically infects 2% of the world population and effective treatment is limited by long duration and significant side-effects. Here, we describe a novel drug, intended...
5.
Maguire A, High K, Auricchio A, Wright J, Pierce E, Testa F, et al.
Lancet . 2009 Oct; 374(9701):1597-605. PMID: 19854499
Background: Gene therapy has the potential to reverse disease or prevent further deterioration of vision in patients with incurable inherited retinal degeneration. We therefore did a phase 1 trial to...
6.
Favaro P, Downey H, Zhou J, Wright J, Hauck B, Mingozzi F, et al.
Mol Ther . 2009 Mar; 17(6):1022-30. PMID: 19293773
The assessment of the risk of germline transmission of vector-coded sequences is critical for clinical translation of gene transfer strategies. We used rabbit models to analyze the risk of germline...
7.
Hauck B, Murphy S, Smith P, Qu G, Liu X, Zelenaia O, et al.
Mol Ther . 2008 Oct; 17(1):144-52. PMID: 18941440
In a gene therapy clinical trial for hemophilia B, adeno-associated virus 2 (AAV2) capsid-specific CD8(+) T cells were previously implicated in the elimination of vector-transduced hepatocytes, resulting in loss of...
8.
Maguire A, Simonelli F, Pierce E, Pugh Jr E, Mingozzi F, Bennicelli J, et al.
N Engl J Med . 2008 Apr; 358(21):2240-8. PMID: 18441370
Leber's congenital amaurosis (LCA) is a group of inherited blinding diseases with onset during childhood. One form of the disease, LCA2, is caused by mutations in the retinal pigment epithelium-specific...
9.
Bennicelli J, Wright J, Komaromy A, Jacobs J, Hauck B, Zelenaia O, et al.
Mol Ther . 2008 Jan; 16(3):458-65. PMID: 18209734
We evaluated the safety and efficacy of an optimized adeno-associated virus (AAV; AAV2.RPE65) in animal models of the RPE65 form of Leber congenital amaurosis (LCA). Protein expression was optimized by...
10.
Wang J, Xie J, Lu H, Chen L, Hauck B, Samulski R, et al.
Proc Natl Acad Sci U S A . 2007 Aug; 104(32):13104-9. PMID: 17664425
Previous studies have documented that 0.1 approximately 1% of input recombinant adeno-associated virus (rAAV) vectors could be stabilized and lead to transgene expression. To characterize the steps involving massive AAV...