Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My NCBI Filters

Text availability

Article attribute

Article type

Publication date

Search Results

1,325 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype.
Weber L, Frati G, Felix T, Hardouin G, Casini A, Wollenschlaeger C, Meneghini V, Masson C, De Cian A, Chalumeau A, Mavilio F, Amendola M, Andre-Schmutz I, Cereseto A, El Nemer W, Concordet JP, Giovannangeli C, Cavazzana M, Miccio A. Weber L, et al. Sci Adv. 2020 Feb 12;6(7):eaay9392. doi: 10.1126/sciadv.aay9392. Print 2020 Feb. Sci Adv. 2020. PMID: 32917636 Free PMC article.
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus.
Antoniani C, Meneghini V, Lattanzi A, Felix T, Romano O, Magrin E, Weber L, Pavani G, El Hoss S, Kurita R, Nakamura Y, Cradick TJ, Lundberg AS, Porteus M, Amendola M, El Nemer W, Cavazzana M, Mavilio F, Miccio A. Antoniani C, et al. Among authors: weber l. Blood. 2018 Apr 26;131(17):1960-1973. doi: 10.1182/blood-2017-10-811505. Epub 2018 Mar 8. Blood. 2018. PMID: 29519807 Free article.
An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype.
Weber L, Poletti V, Magrin E, Antoniani C, Martin S, Bayard C, Sadek H, Felix T, Meneghini V, Antoniou MN, El-Nemer W, Mavilio F, Cavazzana M, Andre-Schmutz I, Miccio A. Weber L, et al. Mol Ther Methods Clin Dev. 2018 Aug 4;10:268-280. doi: 10.1016/j.omtm.2018.07.012. eCollection 2018 Sep 21. Mol Ther Methods Clin Dev. 2018. PMID: 30140714 Free PMC article.
Gene Therapy in a Patient with Sickle Cell Disease.
Ribeil JA, Hacein-Bey-Abina S, Payen E, Magnani A, Semeraro M, Magrin E, Caccavelli L, Neven B, Bourget P, El Nemer W, Bartolucci P, Weber L, Puy H, Meritet JF, Grevent D, Beuzard Y, Chrétien S, Lefebvre T, Ross RW, Negre O, Veres G, Sandler L, Soni S, de Montalembert M, Blanche S, Leboulch P, Cavazzana M. Ribeil JA, et al. Among authors: weber l. N Engl J Med. 2017 Mar 2;376(9):848-855. doi: 10.1056/NEJMoa1609677. N Engl J Med. 2017. PMID: 28249145 Free article.
Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion.
Lagresle-Peyrou C, Lefrère F, Magrin E, Ribeil JA, Romano O, Weber L, Magnani A, Sadek H, Plantier C, Gabrion A, Ternaux B, Félix T, Couzin C, Stanislas A, Tréluyer JM, Lamhaut L, Joseph L, Delville M, Miccio A, André-Schmutz I, Cavazzana M. Lagresle-Peyrou C, et al. Among authors: weber l. Haematologica. 2018 May;103(5):778-786. doi: 10.3324/haematol.2017.184788. Epub 2018 Feb 22. Haematologica. 2018. PMID: 29472357 Free PMC article. Clinical Trial.
1,325 results