Tento výsledek nelze pro nepřihlášené uživatele zobrazit.
K zobrazení výsledku je třeba se přihlásit.

GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a CRISPR base editing therapy.

Autor: Kasiewicz LN; Verve Therapeutics, 201 Brookline Avenue, Suite 601, Boston, MA, 02215, USA., Biswas S; Verve Therapeutics, 201 Brookline Avenue, Suite 601, Boston, MA, 02215, USA., Beach A; Verve Therapeutics, 201 Brookline Avenue, Suite 601, Boston, MA, 02215, USA., Ren H; Verve Therapeutics, 201 Brookline Avenue, Suite 601, Boston, MA, 02215, USA., Dutta C; Verve Therapeutics, 201 Brookline Avenue, Suite 601, Boston, MA, 02215, USA., Mazzola AM; Verve Therapeutics, 201 Brookline Avenue, Suite 601, Boston, MA, 02215, USA., Rohde E; Verve Therapeutics, 201 Brookline Avenue, Suite 601, Boston, MA, 02215, USA., Chadwick A; Verve Therapeutics, 201 Brookline Avenue, Suite 601, Boston, MA, 02215, USA., Cheng C; Verve Therapeutics, 201 Brookline Avenue, Suite 601, Boston, MA, 02215, USA., Garcia SP; Verve Therapeutics, 201 Brookline Avenue, Suite 601, Boston, MA, 02215, USA., Iyer S; Verve Therapeutics, 201 Brookline Avenue, Suite 601, Boston, MA, 02215, USA., Matsumoto Y; Verve Therapeutics, 201 Brookline Avenue, Suite 601, Boston, MA, 02215, USA., Khera AV; Verve Therapeutics, 201 Brookline Avenue, Suite 601, Boston, MA, 02215, USA., Musunuru K; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA., Kathiresan S; Verve Therapeutics, 201 Brookline Avenue, Suite 601, Boston, MA, 02215, USA., Malyala P; Verve Therapeutics, 201 Brookline Avenue, Suite 601, Boston, MA, 02215, USA., Rajeev KG; Verve Therapeutics, 201 Brookline Avenue, Suite 601, Boston, MA, 02215, USA., Bellinger AM; Verve Therapeutics, 201 Brookline Avenue, Suite 601, Boston, MA, 02215, USA. abellinger@vervetx.com.

Publikováno v: Nature communications [Nat Commun] 2023 May 15; Vol. 14 (1), pp. 2776. Date of Electronic Publication: 2023 May 15.

In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates.

Autor: Musunuru K; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA., Chadwick AC; Verve Therapeutics, Cambridge, MA, USA., Mizoguchi T; Verve Therapeutics, Cambridge, MA, USA., Garcia SP; Verve Therapeutics, Cambridge, MA, USA., DeNizio JE; Verve Therapeutics, Cambridge, MA, USA., Reiss CW; Verve Therapeutics, Cambridge, MA, USA., Wang K; Verve Therapeutics, Cambridge, MA, USA., Iyer S; Verve Therapeutics, Cambridge, MA, USA., Dutta C; Verve Therapeutics, Cambridge, MA, USA., Clendaniel V; Verve Therapeutics, Cambridge, MA, USA., Amaonye M; Verve Therapeutics, Cambridge, MA, USA., Beach A; Verve Therapeutics, Cambridge, MA, USA., Berth K; Verve Therapeutics, Cambridge, MA, USA., Biswas S; Verve Therapeutics, Cambridge, MA, USA., Braun MC; Verve Therapeutics, Cambridge, MA, USA., Chen HM; Verve Therapeutics, Cambridge, MA, USA., Colace TV; Verve Therapeutics, Cambridge, MA, USA., Ganey JD; Verve Therapeutics, Cambridge, MA, USA., Gangopadhyay SA; Verve Therapeutics, Cambridge, MA, USA., Garrity R; Verve Therapeutics, Cambridge, MA, USA., Kasiewicz LN; Verve Therapeutics, Cambridge, MA, USA., Lavoie J; Verve Therapeutics, Cambridge, MA, USA., Madsen JA; Verve Therapeutics, Cambridge, MA, USA., Matsumoto Y; Verve Therapeutics, Cambridge, MA, USA., Mazzola AM; Verve Therapeutics, Cambridge, MA, USA., Nasrullah YS; Verve Therapeutics, Cambridge, MA, USA., Nneji J; Verve Therapeutics, Cambridge, MA, USA., Ren H; Verve Therapeutics, Cambridge, MA, USA., Sanjeev A; Verve Therapeutics, Cambridge, MA, USA., Shay M; Verve Therapeutics, Cambridge, MA, USA., Stahley MR; Verve Therapeutics, Cambridge, MA, USA., Fan SHY; Acuitas Therapeutics, Vancouver, British Columbia, Canada., Tam YK; Acuitas Therapeutics, Vancouver, British Columbia, Canada., Gaudelli NM; Beam Therapeutics, Cambridge, MA, USA., Ciaramella G; Beam Therapeutics, Cambridge, MA, USA., Stolz LE; Verve Therapeutics, Cambridge, MA, USA., Malyala P; Verve Therapeutics, Cambridge, MA, USA., Cheng CJ; Verve Therapeutics, Cambridge, MA, USA., Rajeev KG; Verve Therapeutics, Cambridge, MA, USA., Rohde E; Verve Therapeutics, Cambridge, MA, USA., Bellinger AM; Verve Therapeutics, Cambridge, MA, USA., Kathiresan S; Verve Therapeutics, Cambridge, MA, USA. skathiresan@vervetx.com.

Publikováno v: Nature [Nature] 2021 May; Vol. 593 (7859), pp. 429-434. Date of Electronic Publication: 2021 May 19.

A gastric resident drug delivery system for prolonged gram-level dosing of tuberculosis treatment.

Autor: Verma M; Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Tata Center for Technology and Design, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Vishwanath K; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Eweje F; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Mechanical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Division of Gastroenterology, Hepatology, and Endoscopy, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA., Roxhed N; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Micro and Nanosystems, KTH Royal Institute of Technology, Stockholm 10044, Sweden., Grant T; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Castaneda M; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Steiger C; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Division of Gastroenterology, Hepatology, and Endoscopy, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA.; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Mazdiyasni H; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Bensel T; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Minahan D; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Soares V; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Salama JAF; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Lopes A; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Hess K; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Cleveland C; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Fulop DJ; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Hayward A; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Division of Comparative Medicine, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Collins J; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Tamang SM; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Hua T; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Ikeanyi C; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Mechanical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Zeidman G; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Mechanical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Mule E; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Boominathan S; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Electrical Engineering and Computer Science, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Popova E; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Biology, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Miller JB; Department of Mechanical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Sloan School of Management, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Bellinger AM; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Cardiovascular Division, Department of Medicine, Harvard Medical School, Boston, MA 02115, USA., Collins D; Management Sciences for Health, Medford, MA 02155, USA.; Boston University School of Public Health, Boston, MA 02118, USA., Leibowitz D; Tata Center for Technology and Design, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Mechanical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Batra S; Operation ASHA, New Delhi 110076, India., Ahuja S; Operation ASHA, New Delhi 110076, India., Bajiya M; Operation ASHA, New Delhi 110076, India., Batra S; Operation ASHA, New Delhi 110076, India., Sarin R; National Institute of Tuberculosis and Respiratory Diseases, New Delhi 110030, India., Agarwal U; National Institute of Tuberculosis and Respiratory Diseases, New Delhi 110030, India., Khaparde SD; Former Deputy Director General and Head of Central TB Division, Government of India, New Delhi 110011, India., Gupta NK; Department of Respiratory Medicine, Safdarjung Hospital, New Delhi 110029, India., Gupta D; Division of Pulmonary and Critical Care Medicine, All India Institute of Medical Sciences, New Delhi 110029, India., Bhatnagar AK; Rajan Babu Institute for Pulmonary Medicine and Tuberculosis, New Delhi 110009, India., Chopra KK; New Delhi Tuberculosis Centre, New Delhi 110002, India., Sharma N; Department of Community Medicine, Maulana Azad Medical College, New Delhi 110002, India., Khanna A; Lok Nayak Hospital Chest Clinic, New Delhi 110002, India., Chowdhury J; Tata Trusts, Mumbai 400001, India., Stoner R; Tata Center for Technology and Design, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; MIT Energy Initiative, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Slocum AH; Tata Center for Technology and Design, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Mechanical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Cima MJ; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Tata Center for Technology and Design, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Materials Science and Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Furin J; Department of Global Health and Social Medicine, Harvard Medical School, Boston, MA 02115, USA., Langer R; Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA. rlanger@mit.edu ctraverso@bwh.harvard.edu cgt20@mit.edu.; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Tata Center for Technology and Design, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Mechanical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Institute for Medical Engineering and Science, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Media Lab, Massachusetts Institute of Technology, Cambridge, MA 02139, USA., Traverso G; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA. rlanger@mit.edu ctraverso@bwh.harvard.edu cgt20@mit.edu.; Tata Center for Technology and Design, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Department of Mechanical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.; Division of Gastroenterology, Hepatology, and Endoscopy, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA.; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.

Publikováno v: Science translational medicine [Sci Transl Med] 2019 Mar 13; Vol. 11 (483).

Development of an oral once-weekly drug delivery system for HIV antiretroviral therapy.

Autor: Kirtane AR; Department of Chemical Engineering and David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA., Abouzid O; Department of Chemical Engineering and David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.; Département Biosciences, Institut National des Sciences Appliquées de Lyon, 20 Avenue Albert Einstein, Villeurbanne, France, 69100., Minahan D; Department of Chemical Engineering and David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA., Bensel T; Department of Chemical Engineering and David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA., Hill AL; Program for Evolutionary Dynamics, Harvard University, Cambridge, Massachusetts, 02138, USA., Selinger C; Institute for Disease Modeling, Bellevue, WA, 98005, USA., Bershteyn A; Institute for Disease Modeling, Bellevue, WA, 98005, USA., Craig M; Program for Evolutionary Dynamics, Harvard University, Cambridge, Massachusetts, 02138, USA., Mo SS; Program for Evolutionary Dynamics, Harvard University, Cambridge, Massachusetts, 02138, USA., Mazdiyasni H; Department of Chemical Engineering and David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA., Cleveland C; Department of Chemical Engineering and David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA.; Division of Gastroenterology Brigham and Women's Hospital, Harvard Medical School, Boston, MA, 02115, USA., Rogner J; Department of Chemical Engineering and David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA., Lee YL; Department of Chemical Engineering and David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA., Booth L; Department of Chemical Engineering and David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA., Javid F, Wu SJ; Department of Mechanical Engineering, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA., Grant T; Lyndra Inc, Watertown, MA, 02472, USA., Bellinger AM; Lyndra Inc, Watertown, MA, 02472, USA., Nikolic B; Biomatics Capital, 1107 1st Avenue, Apartment 1305, Seattle, WA, 98101, USA., Hayward A; Department of Chemical Engineering and David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA., Wood L; Institute for Disease Modeling, Bellevue, WA, 98005, USA., Eckhoff PA; Institute for Disease Modeling, Bellevue, WA, 98005, USA., Nowak MA; Program for Evolutionary Dynamics, Harvard University, Cambridge, Massachusetts, 02138, USA., Langer R; Department of Chemical Engineering and David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA. rlanger@mit.edu.; Media Lab, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA. rlanger@mit.edu.; Institute for Medical Engineering and Science, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA. rlanger@mit.edu., Traverso G; Department of Chemical Engineering and David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA. ctraverso@bwh.harvard.edu.; Division of Gastroenterology Brigham and Women's Hospital, Harvard Medical School, Boston, MA, 02115, USA. ctraverso@bwh.harvard.edu.

Publikováno v: Nature communications [Nat Commun] 2018 Jan 09; Vol. 9 (1), pp. 2. Date of Electronic Publication: 2018 Jan 09.