Journal

bioRxiv : the preprint server for biology
Titre abrégé: bioRxiv
Pays: United States
ID NLM: 101680187

Informations de publication

Date de publication:
29 Jul 2023
Historique:
pubmed: 7 8 2023
medline: 7 8 2023
entrez: 7 8 2023
Statut: epublish

Résumé

Homology Directed Repair (HDR)-based genome editing is an approach that could permanently correct a broad range of genetic diseases. However, its utility is limited by inefficient and imprecise DNA repair mechanisms in terminally differentiated tissues. Here, we tested "Repair Drive", a novel method for improving targeted gene insertion in the liver by selectively expanding correctly repaired hepatocytes

Identifiants

pubmed: 37546995
doi: 10.1101/2023.07.26.550728
pmc: PMC10402145
pii:
doi:

Types de publication

Preprint

Langues

eng

Subventions

Organisme : NHLBI NIH HHS
ID : UG3 HL151545
Pays : United States
Organisme : NHLBI NIH HHS
ID : R01 HL152314
Pays : United States
Organisme : NIDDK NIH HHS
ID : R56 DK115461
Pays : United States
Organisme : NHLBI NIH HHS
ID : R01 HL132840
Pays : United States
Organisme : NCI NIH HHS
ID : P30 CA125123
Pays : United States
Organisme : NIDDK NIH HHS
ID : P30 DK056338
Pays : United States
Organisme : NIH HHS
ID : U42 OD026645
Pays : United States
Organisme : NIDDK NIH HHS
ID : R01 DK124477
Pays : United States
Organisme : NIDDK NIH HHS
ID : R01 DK115461
Pays : United States

Déclaration de conflit d'intérêts

Competing Interests. W.R.L., A.H., K.D.B., M.D.G., and M.N.F. have filed a patent application for the Repair Drive technology and its application to gene therapy.

Auteurs

Marco De Giorgi (M)

Department of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.

So Hyun Park (SH)

Department of Bioengineering, Rice University, Houston, TX 77030, USA.

Adam Castoreno (A)

Alnylam Pharmaceuticals Inc, 675 W Kendall St, Cambridge, MA 02142, USA.

Mingming Cao (M)

Department of Bioengineering, Rice University, Houston, TX 77030, USA.

Ayrea Hurley (A)

Department of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.

Lavanya Saxena (L)

Department of Bioengineering, Rice University, Houston, TX 77030, USA.

Marcel A Chuecos (MA)

Department of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.
Translational Biology and Molecular Medicine Program, Baylor College of Medicine, Houston, TX 77030, USA.

Christopher J Walkey (CJ)

Department of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.

Alexandria M Doerfler (AM)

Department of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.

Mia N Furgurson (MN)

Department of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.

M Cecilia Ljungberg (MC)

Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
Duncan Neurological Research Institute, Texas Children's Hospital, Houston, TX 77030, USA.

Kalyani R Patel (KR)

Department of Pathology, Texas Children's Hospital, Houston, TX 77030, USA.

Sarah Hyde (S)

Alnylam Pharmaceuticals Inc, 675 W Kendall St, Cambridge, MA 02142, USA.

Tyler Chickering (T)

Alnylam Pharmaceuticals Inc, 675 W Kendall St, Cambridge, MA 02142, USA.

Stephanie Lefebvre (S)

Alnylam Pharmaceuticals Inc, 675 W Kendall St, Cambridge, MA 02142, USA.

Kelly Wassarman (K)

Alnylam Pharmaceuticals Inc, 675 W Kendall St, Cambridge, MA 02142, USA.

Patrick Miller (P)

Alnylam Pharmaceuticals Inc, 675 W Kendall St, Cambridge, MA 02142, USA.

June Qin (J)

Alnylam Pharmaceuticals Inc, 675 W Kendall St, Cambridge, MA 02142, USA.

Mark K Schlegel (MK)

Alnylam Pharmaceuticals Inc, 675 W Kendall St, Cambridge, MA 02142, USA.

Ivan Zlatev (I)

Alnylam Pharmaceuticals Inc, 675 W Kendall St, Cambridge, MA 02142, USA.

Rich Gang Li (RG)

Department of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.
Texas Heart Institute, Houston, TX 77030, USA.

Jong Kim (J)

Department of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.
Texas Heart Institute, Houston, TX 77030, USA.

James F Martin (JF)

Department of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.
Texas Heart Institute, Houston, TX 77030, USA.

Karl-Dimiter Bissig (KD)

Department of Pediatrics, Alice and Y. T. Chen Center for Genetics and Genomics, Division of Medical Genetics, Duke University, Durham, NC 27710, USA.

Vasant Jadhav (V)

Alnylam Pharmaceuticals Inc, 675 W Kendall St, Cambridge, MA 02142, USA.

Gang Bao (G)

Department of Bioengineering, Rice University, Houston, TX 77030, USA.

William R Lagor (WR)

Department of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.

Classifications MeSH