Ready for repair? Gene editing enters the clinic for the treatment of human disease

MPT Ernst, M Broeders, P Herrero-Hernandez… - … Therapy Methods & …, 2020 - cell.com
We present an overview of clinical trials involving gene editing using clustered interspaced
short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription …

Immunological barriers to haematopoietic stem cell gene therapy

CT Charlesworth, I Hsu, AC Wilkinson… - Nature Reviews …, 2022 - nature.com
Cell and gene therapies using haematopoietic stem cells (HSCs) epitomize the
transformative potential of regenerative medicine. Recent clinical successes for gene …

In vivo engineered B cells secrete high titers of broadly neutralizing anti-HIV antibodies in mice

AD Nahmad, CR Lazzarotto, N Zelikson, T Kustin… - Nature …, 2022 - nature.com
Transplantation of B cells engineered ex vivo to secrete broadly neutralizing antibodies
(bNAbs) has shown efficacy in disease models. However, clinical translation of this …

Vaccine elicitation of HIV broadly neutralizing antibodies from engineered B cells

D Huang, JT Tran, A Olson, T Vollbrecht… - Nature …, 2020 - nature.com
HIV broadly neutralizing antibodies (bnAbs) can suppress viremia and protect against HIV
infection. However, their elicitation is made difficult by low frequencies of appropriate …

B cells engineered to express pathogen-specific antibodies protect against infection

HF Moffett, CK Harms, KS Fitzpatrick, MR Tooley… - Science …, 2019 - science.org
Effective vaccines inducing lifelong protection against many important infections such as
respiratory syncytial virus (RSV), HIV, influenza virus, and Epstein-Barr virus (EBV) are not …

Engineered B cells expressing an anti-HIV antibody enable memory retention, isotype switching and clonal expansion

AD Nahmad, Y Raviv, M Horovitz-Fried, I Sofer… - Nature …, 2020 - nature.com
ABSTRACT HIV viremia can be controlled by chronic antiretroviral therapy. As a potentially
single-shot alternative, B cells engineered by CRISPR/Cas9 to express anti-HIV broadly …

Ex vivo engineered human plasma cells exhibit robust protein secretion and long-term engraftment in vivo

RYH Cheng, KL Hung, T Zhang, CM Stoffers… - Nature …, 2022 - nature.com
Due to their unique longevity and capacity to secrete high levels of protein, plasma B cells
have the potential to be used as a cell therapy for protein replacement. Here, we show that …

Enhancing antibody responses by multivalent antigen display on thymus-independent DNA origami scaffolds

EC Wamhoff, L Ronsard, J Feldman… - Nature …, 2024 - nature.com
Protein-based virus-like particles (P-VLPs) are commonly used to spatially organize
antigens and enhance humoral immunity through multivalent antigen display. However, P …

CDKN1A regulates Langerhans cell survival and promotes Treg cell generation upon exposure to ionizing irradiation

JG Price, J Idoyaga, H Salmon, B Hogstad… - Nature …, 2015 - nature.com
Abstract Treatment with ionizing radiation (IR) can lead to the accumulation of tumor-
infiltrating regulatory T cells (Treg cells) and subsequent resistance of tumors to …

[HTML][HTML] HIV-specific humoral immune responses by CRISPR/Cas9-edited B cells

H Hartweger, AT McGuire, M Horning… - Journal of Experimental …, 2019 - rupress.org
A small number of HIV-1–infected individuals develop broadly neutralizing antibodies to the
virus (bNAbs). These antibodies are protective against infection in animal models. However …