In utero gene editing for monogenic lung disease

Multiplex Genome Engineering Using CRISPR/Cas Systems

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Genome engineering using the CRISPR-Cas9 system

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The new frontier of genome engineering with CRISPR-Cas9

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Cystic Fibrosis

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A global double-fluorescent Cre reporter mouse

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Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements

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Evaluation of off-target and on-target scoring algorithms and integration into the guide RNA selection tool CRISPOR

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Cystic Fibrosis

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Pulmonary drug delivery. Part I: physiological factors affecting therapeutic effectiveness of aerosolized medications.

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Cas9 as a versatile tool for engineering biology

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The delivery challenge: fulfilling the promise of therapeutic genome editing

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Ionizable lipid nanoparticles for in utero mRNA delivery

Riley, R. S., Kashyap, M. V., Billingsley, M. M., White, B., Alameh, M.-G., Bose, S. K., Zoltick, P. W., Li, H., Zhang, R., Cheng, A. Y., et al. (2021) · Science Advances · doi:10.1126/sciadv.aba1028 · Cited by 222

Lung and liver editing by lipid nanoparticle delivery of a stable CRISPR–Cas9 ribonucleoprotein

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Applications and challenges of CRISPR-Cas gene-editing to disease treatment in clinics

Liu, W., Li, L., Jiang, J., Wu, M., & Lin, P. (2021) · Precision Clinical Medicine · doi:10.1093/pcmedi/pbab014 · Cited by 196

Strategies to overcome the main challenges of the use of CRISPR/Cas9 as a replacement for cancer therapy

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Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases

Wu, S., Li, Q.-C., Yin, C., Xue, W., & Song, C. (2020) · Theranostics · doi:10.7150/thno.43360 · Cited by 145

Patient-specific iPSCs carrying an SFTPC mutation reveal the intrinsic alveolar epithelial dysfunction at the inception of interstitial lung disease

Alysandratos, K., Russo, S. J., Petcherski, A., Taddeo, E. P., Acín‐Pérez, R., Villacorta-Martín, C., Jean, J., Mulugeta, S., Rodríguez, L. R., Blum, B. C., et al. (2021) · Cell Reports · doi:10.1016/j.celrep.2021.109636 · Cited by 116

Carrier strategies boost the application of CRISPR/Cas system in gene therapy

Xu, Z., Wang, Q., Zhong, H., Jiang, Y., Shi, X., Yuan, B., Yu, N., Zhang, S., Yuan, X., Guo, S., et al. (2022) · Exploration · doi:10.1002/exp.20210081 · Cited by 105

Ionizable Lipid Nanoparticles for Therapeutic Base Editing of Congenital Brain Disease

Palanki, R., Bose, S. K., Dave, A., White, B. M., Berkowitz, C., Luks, V., Yaqoob, F., Han, E., Swingle, K. L., Menon, P., et al. (2023) · ACS Nano · doi:10.1021/acsnano.3c02268 · Cited by 77

Delivery technologies for in utero gene therapy

Palanki, R., Peranteau, W. H., & Mitchell, M. J. (2021) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.11.002 · Cited by 64

Amniotic fluid stabilized lipid nanoparticles for in utero intra-amniotic mRNA delivery

Swingle, K. L., Billingsley, M. M., Bose, S. K., White, B., Palanki, R., Dave, A., Patel, S. K., Gong, N., Hamilton, A. G., Alameh, M.-G., et al. (2022) · Journal of Controlled Release · doi:10.1016/j.jconrel.2021.10.031 · Cited by 64

Delivery technologies for women’s health applications

Swingle, K. L., Ricciardi, A. S., Peranteau, W. H., & Mitchell, M. J. (2023) · Nature Reviews Bioengineering · doi:10.1038/s44222-023-00040-w · Cited by 64

Lung repair and regeneration: Advanced models and insights into human disease

Basil, M. C., Alysandratos, K., Kotton, D. N., & Morrisey, E. E. (2024) · Cell stem cell · doi:10.1016/j.stem.2024.02.009 · Cited by 65

Exploiting the placenta for nanoparticle-mediated drug delivery during pregnancy

Figueroa‐Espada, C. G., Hofbauer, S. I., Mitchell, M. J., & Riley, R. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.09.006 · Cited by 65

Targeting Alveolar Repair in Idiopathic Pulmonary Fibrosis

Ptasinski, V., Stegmayr, J., Belvisi, M. G., Wagner, D. E., & Murray, L. A. (2021) · American Journal of Respiratory Cell and Molecular Biology · doi:10.1165/rcmb.2020-0476tr · Cited by 60

Lipid- and polymer-based nanoparticle systems for the delivery of CRISPR/Cas9

Ashok, B., Peppas, N. A., & Wechsler, M. E. (2021) · Journal of Drug Delivery Science and Technology · doi:10.1016/j.jddst.2021.102728 · Cited by 62

In vivo delivery of CRISPR-Cas9 genome editing components for therapeutic applications

Huang, K. L., Zapata, D. A., Tang, Y., Teng, Y., & Li, Y. (2022) · Biomaterials · doi:10.1016/j.biomaterials.2022.121876 · Cited by 61

In utero delivery of targeted ionizable lipid nanoparticles facilitates in vivo gene editing of hematopoietic stem cells

Palanki, R., Riley, J. S., Bose, S. K., Luks, V. L., Dave, A., Kus, N. J., White, B. M., Ricciardi, A. S., Swingle, K. L., Xue, L., et al. (2024) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2400783121 · Cited by 50

Yeast Cell wall Particle mediated Nanotube-RNA delivery system loaded with miR365 Antagomir for Post-traumatic Osteoarthritis Therapy via Oral Route

Zhang, L., Peng, H., Zhang, W., Li, Y., Liu, L., & Leng, T. (2020) · Theranostics · doi:10.7150/thno.46761 · Cited by 51

The Future of In Utero Gene Therapy

Peranteau, W. H., & Flake, A. W. (2020) · Molecular Diagnosis & Therapy · doi:10.1007/s40291-020-00445-y · Cited by 47

Applications of CRISPR as a potential therapeutic

Luthra, R., Kaur, S. P., & Bhandari, K. (2021) · Life Sciences · doi:10.1016/j.lfs.2021.119908 · Cited by 48

Pulmonary Consequences of Prenatal Inflammatory Exposures: Clinical Perspective and Review of Basic Immunological Mechanisms

Jackson, C. M., Mukherjee, S., Wilburn, A. N., Cates, C., Lewkowich, I. P., Deshmukh, H. S., Zacharias, W. J., & Chougnet, C. A. (2020) · Frontiers in Immunology · doi:10.3389/fimmu.2020.01285 · Cited by 45

Gene Therapy Potential for Genetic Disorders of Surfactant Dysfunction

Cooney, A. L., Wambach, J., Sinn, P. L., & McCray, P. B. (2022) · Frontiers in Genome Editing · doi:10.3389/fgeed.2021.785829 · Cited by 42

Women-specific routes of administration for drugs: A critical overview

das Neves, J., Notario-Pérez, F., & Sarmento, B. F. C. C. (2021) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2021.113865 · Cited by 44

Recent Advances and Future Perspectives of In Vivo Targeted Delivery of Genome-Editing Reagents to Germ cells, Embryos, and Fetuses in Mice

Sato, M., Takabayashi, S., Akasaka, E., & Nakamura, S. (2020) · Cells · doi:10.3390/cells9040799 · Cited by 40

Correction of Airway Stem Cells: Genome Editing Approaches for the Treatment of Cystic Fibrosis

King, N. E., Suzuki, S., Barillà, C., Hawkins, F. J., Randell, S. H., Reynolds, S. D., Stripp, B. R., & Davis, B. R. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.160 · Cited by 40

CRISPR/Cas-Dependent and Nuclease-Free In Vivo Therapeutic Gene Editing

Dasgupta, I., Flotte, T. R., & Keeler, A. M. (2021) · Human Gene Therapy · doi:10.1089/hum.2021.013 · Cited by 38

Targeted drug delivery for maternal and perinatal health: Challenges and opportunities

Sharma, A., Sah, N., Kannan, S., & Kannan, R. M. (2021) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2021.113950 · Cited by 35

Novel Insights into the Therapeutic Potential of Lung-Targeted Gene Transfer in the Most Common Respiratory Diseases

Bisserier, M., Sun, X., Fazal, S., Turnbull, I. C., Bonnet, S., & Hadri, L. (2022) · Cells · doi:10.3390/cells11060984 · Cited by 36

In utero delivery of mRNA to the heart, diaphragm and muscle with lipid nanoparticles

Gao, K., Li, J., Song, H., Han, H., Wang, Y., Yin, B., Farmer, D. L., Murthy, N., & Wang, A. (2023) · Bioactive Materials · doi:10.1016/j.bioactmat.2023.02.011 · Cited by 33

Design Approaches for Generating Organ Constructs

Xia, Y. F., & Belmonte, J. C. I. (2019) · Cell stem cell · doi:10.1016/j.stem.2019.05.016 · Cited by 32

In Utero Gene Therapy: Progress and Challenges

Bose, S. K., Menon, P. V., & Peranteau, W. H. (2021) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2021.05.007 · Cited by 29

Transplacental Gene Delivery (TPGD) as a Noninvasive Tool for Fetal Gene Manipulation in Mice

Nakamura, S., Watanabe, S., Ando, N., Ishihara, M., & Sato, M. (2019) · International Journal of Molecular Sciences · doi:10.3390/ijms20235926 · Cited by 26

Targeted regulation of fibroblast state by CRISPR-mediated CEBPA expression

Liu, W. Q., Meridew, J. A., Aravamudhan, A., Ligresti, G., Tschumperlin, D. J., & Tan, Q. (2019) · Respiratory Research · doi:10.1186/s12931-019-1253-1 · Cited by 26

Pulmonary immune cell transcriptome changes in double-hit model of BPD induced by chorioamnionitis and postnatal hyperoxia

Shrestha, D., Ye, G. X., Stabley, D. L., Betal, S. G., Zhu, Y. Y., Glazewski, L., Holbrook, J., Sethi, M., Hesek, A. M., Shaffer, T. H., et al. (2021) · Pediatric Research · doi:10.1038/s41390-020-01319-z · Cited by 25

Nanoparticles for delivery of agents to fetal lungs

Ullrich, S. J., Freedman-Weiss, M. R., Ahle, S. L., Mandl, H. K., Piotrowski-Daspit, A. S., Roberts, K., Yung, N. K., Maassel, N. L., Bauer-Pisani, T. J., Ricciardi, A. S., et al. (2021) · Acta Biomaterialia · doi:10.1016/j.actbio.2021.01.024 · Cited by 25

Alveolar Epithelial Cell Dysfunction in Acute Respiratory Distress Syndrome: Mechanistic Insights and Targeted Interventions

Wang, J., & Chao, J. (2025) · Biomedicines · doi:10.3390/biomedicines13092299 · Cited by 25

Intrauterine Hypoxia and Epigenetic Programming in Lung Development and Disease

Tong, Y., Zhang, S., Riddle, S. R., Zhang, L., Song, R., & Yue, D. (2021) · Biomedicines · doi:10.3390/biomedicines9080944 · Cited by 24

Embryo and fetal gene editing: Technical challenges and progress toward clinical applications

Mattar, C. N. Z., Chew, W. L., & Lai, P. S. (2024) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2024.101229 · Cited by 25

Human pluripotent stem cell modeling of alveolar type 2 cell dysfunction caused by ABCA3 mutations

Sun, Y. L., Hennessey, E., Heins, H. B., Yang, P., Villacorta-Martín, C., Kwan, J., Gopalan, K., James, M. F., Emili, A., Cole, F. S., et al. (2024) · Journal of Clinical Investigation · doi:10.1172/jci164274 · Cited by 23

Prenatal Somatic Cell Gene Therapies: Charting a Path Toward Clinical Applications (Proceedings of the CERSI‐FDA Meeting)

Herzeg, A., Almeida-Porada, G. D., Charo, R. A., David, A. L., Gonzalez‐Velez, J. M., Gupta, N. M., Lapteva, L., Lianoglou, B. R., Peranteau, W. H., Porada, C. D., et al. (2022) · The Journal of Clinical Pharmacology · doi:10.1002/jcph.2127 · Cited by 24

Gene modification therapies for hereditary diseases in the fetus

Mattar, C. N. Z., Chan, J. K. Y., & Choolani, M. A. (2023) · Prenatal Diagnosis · doi:10.1002/pd.6347 · Cited by 23

Gene Therapeutics for Surfactant Dysfunction Disorders: Targeting the Alveolar Type 2 Epithelial Cell

Sitaraman, S., Alysandratos, K., Wambach, J., & Limberis, M. P. (2022) · Human Gene Therapy · doi:10.1089/hum.2022.130 · Cited by 22

Towards personalized therapies for genetic disorders of surfactant dysfunction

de Nieuwburgh, M. P., Wambach, J., Griese, M., & Danhaive, O. (2023) · Seminars in Fetal and Neonatal Medicine · doi:10.1016/j.siny.2023.101500 · Cited by 22

Gene and Stem Cell Therapies for Fetal Care

O’Connell, A. E., Guseh, S. H., Lapteva, L., Cummings, C. L., Wilkins-Haug, L. E., Chan, J. K. Y., Peranteau, W. H., Almeida-Porada, G. D., & Kourembanas, S. (2020) · Archives of Pediatrics and Adolescent Medicine · doi:10.1001/jamapediatrics.2020.1519 · Cited by 20

Society for Maternal-Fetal Medicine Special Statement: Beyond the scalpel: in utero fetal gene therapy and curative medicine

Shanahan, M. A., Aagaard, K. M., McCullough, L. B., Chervenak, F. A., & Shamshirsaz, A. A. (2021) · American Journal of Obstetrics and Gynecology · doi:10.1016/j.ajog.2021.09.001 · Cited by 22

Prenatal Gene Therapy

Schwab, M. E., & MacKenzie, T. C. (2021) · Clinical Obstetrics & Gynecology · doi:10.1097/grf.0000000000000655 · Cited by 20

Efficient Adeno-associated Virus–mediated Transgenesis in Alveolar Stem Cells and Associated Niches

Konkimalla, A., Elmore, Z. C., Konishi, S., Macadlo, L., Katsura, H., Tata, A., Asokan, A., & Tata, P. R. (2023) · American Journal of Respiratory Cell and Molecular Biology · doi:10.1165/rcmb.2022-0424ma · Cited by 20

Beliefs and Values About Gene Therapy and In-Utero Gene Editing in Patients with Hemophilia and Their Relatives

Vasquez-Loarte, T. C., Lucas, T. L., Harris-Wai, J. N., & Bowen, D. J. (2020) · Patient · doi:10.1007/s40271-020-00442-7 · Cited by 17

New Directions in Pulmonary Gene Therapy

Vu, A., & McCray, P. B. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.166 · Cited by 19

Addressing the dark matter of gene therapy: technical and ethical barriers to clinical application

Kratzer, K., Getz, L. J., Peterlini, T., Masson, J., & Dellaire, G. (2021) · Human Genetics · doi:10.1007/s00439-021-02272-5 · Cited by 14

Hematogenous Routing of Exogenous Messenger RNA Delivered Into the Amniotic Fluid

Moskowitzova, K., Whitlock, A. E., Zurakowski, D., & Fauza, D. O. (2023) · Journal of Surgical Research · doi:10.1016/j.jss.2023.03.037 · Cited by 12

Genome Editing for Rare Diseases

Pradhan, A., Kalin, T. V., & Kalinichenko, V. V. (2020) · Current Stem Cell Reports · doi:10.1007/s40778-020-00175-1 · Cited by 10

In Utero Gene Therapy and its Application in Genetic Hearing Loss

Kong, C., Yin, G., Wang, X., & Sun, Y. (2024) · Advanced Biology · doi:10.1002/adbi.202400193 · Cited by 10

Possible association between SP-C mutations and lung cancer: Two case reports and review of literature

Darawshy, F., Rmeileh, A. A., Kuint, R., & Berkman, N. (2021) · Cancer Treatment and Research Communications · doi:10.1016/j.ctarc.2021.100461 · Cited by 9

Genes in pediatric pulmonary arterial hypertension and the most promising BMPR2 gene therapy

Dai, L., & Du, L. (2022) · Frontiers in Genetics · doi:10.3389/fgene.2022.961848 · Cited by 8

Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies

Lederer, C. W., Koniali, L., Buerki‐Thurnherr, T., Papasavva, P. L., Grutta, S. L., Licari, A., Štaud, F., Bonifazi, D., & Kleanthous, M. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14040793 · Cited by 9

A Fluorescent Reporter Mouse for In Vivo Assessment of Genome Editing with Diverse Cas Nucleases and Prime Editors

Chen, Z., Kwan, S., Mir, A., Hazeltine, M. D., Shin, M., Liang, S., Chan, I. L., Kelly, K. L., Ghanta, K. S., Gaston, N., et al. (2023) · The CRISPR Journal · doi:10.1089/crispr.2023.0048 · Cited by 7

Sulfonium lipid nanoparticles for intranasal mRNA delivery to lung epithelial and immune cells

Men, Y., Popoola, D. O., Cao, Z. L., Li, Y., Wilkens, S., Teng, Y., Meng, Q., Hershenson, M., & Li, Y. (2025) · Acta Biomaterialia · doi:10.1016/j.actbio.2025.08.032 · Cited by 6

Systemic mRNA-LNP administration in fetuses improves survival in a mouse model of spinal muscular atrophy

Carpenter, M. D., Kus, N. J., Choi, R. B., Said, H., Dumitru, A. M., Bauer, J. H., Wang, S., Luks, V. L., Teerdhala, S., Shah, A., et al. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101599 · Cited by 7

Mechanisms of Surfactant Maturation in Development and Disease

Pranty-Weßelmann, A. I., Ocaña, S. C., & Dickens, J. A. (2025) · Physiology · doi:10.1152/physiol.00033.2025 · Cited by 6

Genome editing and cancer therapy: handling the hypoxia-responsive pathway as a promising strategy

Stampone, E., Bencivenga, D., Capellupo, M. C., Roberti, D., Tartaglione, I., Perrotta, S., Ragione, F. D., & Borriello, A. (2023) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-023-04852-2 · Cited by 7

Non‐Viral RNA Delivery During Pregnancy: Opportunities and Challenges

Chaudhary, N., Newby, A. N., & Whitehead, K. A. (2023) · Small · doi:10.1002/smll.202306134 · Cited by 6

Molecular and Cellular In Utero Therapy

Berkowitz, C. L., Luks, V. L., Puc, M., & Peranteau, W. H. (2022) · Clinics in Perinatology · doi:10.1016/j.clp.2022.06.005 · Cited by 6

Foetal genome editing

Bose, S. K., Kennedy, K., & Peranteau, W. H. (2023) · Current Opinion in Obstetrics & Gynecology · doi:10.1097/gco.0000000000000854 · Cited by 5

Advances in applications of the CRISPR/Cas9 system for respiratory diseases

Ajaykumar, C., Rajkumar, S., Suresh, B., Birappa, G., Gowda, D. A. A., Jayachandran, A., Kim, K.-S., Hong, S.-H., & Ramakrishna, S. (2024) · Progress in molecular biology and translational science · doi:10.1016/bs.pmbts.2024.07.014 · Cited by 5

Lung stem cells and therapy for cystic fibrosis

Vaidyanathan, S., McCarra, M. B., & Desai, T. (2021) · European Respiratory Society eBooks · doi:10.1183/2312508x.10010520 · Cited by 5

Prenatal Gene Therapy for Metabolic Disorders

Coons, B. E., & Peranteau, W. H. (2021) · Clinical Obstetrics & Gynecology · doi:10.1097/grf.0000000000000662 · Cited by 5

Advances in CRISPR/Cas-Based Strategies on Zoonosis

Zhang, Y., Peng, Q., Zhang, R., Li, C., Xu, Q., Xia, L., Wang, Y., Liu, P., & Pan, H. (2023) · Transboundary and Emerging Diseases · doi:10.1155/2023/9098445 · Cited by 5

Nonviral CRISPR/Cas9 mutagenesis for streamlined generation of mouse lung cancer models

Lara‐Sáez, I., Mencía, Á., Recuero, E., Li, Y., García, M., Oteo, M., Gallego, M. I., Enguita, A. B., de Prado-Verdún, D., Sigen, A., et al. (2024) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2322917121 · Cited by 5

Intraplacental injection of AAV9-CMV-iCre results in the widespread transduction of multiple organs in double-reporter mouse embryos

Gogoleva, N., Shahri, Z. J., Noda, A., Liao, C.-W., Wakimoto, A., Inoue, Y., Jeon, H., Takahashi, S., & Hamada, M. (2023) · EXPERIMENTAL ANIMALS · doi:10.1538/expanim.23-0044 · Cited by 4

Old and emerging therapies for childhood interstitial lung disease (chILD): a systematic review

Tonazzo, V., Castaldo, R. J., Zanconato, S., Carraro, S., & Ferraro, V. A. (2026) · European Respiratory Review · doi:10.1183/16000617.0062-2025 · Cited by 4

Reporter Mice for Gene Editing: A Key Tool for Advancing Gene Therapy of Rare Diseases

Li, S.-A., & Brakebusch, C. (2024) · Cells · doi:10.3390/cells13171508 · Cited by 4

In Utero Therapies, the Next Frontier

Kolarova, T. R., & Cheng, E. (2025) · American Journal of Medical Genetics Part C Seminars in Medical Genetics · doi:10.1002/ajmg.c.32135 · Cited by 4

Association analysis of the surfactant protein-C gene to childhood asthma

Nefzi, M., Wahabi, I., Fredj, S. H., Othmani, R., Dabboubi, R., Boussetta, K., Fanen, P., & Messaoud, T. B. (2020) · Journal of Asthma · doi:10.1080/02770903.2020.1827419 · Cited by 4

Administration of Drugs/Gene Products to the Respiratory System: A Historical Perspective of the Use of Inert Liquids

Alapati, D., & Shaffer, T. H. (2022) · Frontiers in Physiology · doi:10.3389/fphys.2022.871893 · Cited by 4

Recent Genome-Editing Approaches toward Post-Implanted Fetuses in Mice

Nakamura, S., Inada, E., Saitoh, I., & Sato, M. (2023) · BioTech · doi:10.3390/biotech12020037 · Cited by 4

Engraftment of wild-type alveolar type II epithelial cells in surfactant protein C deficient mice

Predella, C., Lapsley, L. M., Ni, K., Thimraj, T. A., Liu, H., Murray, J. W., Motelow, J. E., Snoeck, H., Glasser, S. W., Saqi, A., et al. (2026) · npj Regenerative Medicine · doi:10.1038/s41536-026-00455-0 · Cited by 4

Fluorescent in vivo editing reporter (FIVER): A novel multispectral reporter of in vivo genome editingPreprint

Tennant, P. A., Foster, R. G., Dodd, D. O., Sou, I. F., McPhie, F., Younger, N. T., Murphy, L. C., Pearson, M. W., Vernay, B., Keighren, M., et al. (2020) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2020.07.14.200170 · Cited by 4

How CRISPR Is Revolutionizing the Generation of New Models for Cancer ResearchPreprint

Rivera, F. J. S., & Dow, L. E. (2023) · Cold Spring Harbor Perspectives in Medicine · doi:10.1101/cshperspect.a041384 · Cited by 4

Briefing of pulmonary sarcoidosis: Reduction-oxidation, misleading and possibilities

Habib, I., Jawed, J. J., Nasrin, T., & Shaikh, S. (2024) · Indian Journal of Tuberculosis · doi:10.1016/j.ijtb.2024.07.003 · Cited by 3

In Utero Gene Therapy for Sickle Cell Disease: Current Evidence, Ethical Considerations, and Future Directions—A Scoping Review

Cudjoe, E., Thorsen, M. M., Molokwu, N., & Russo, M. (2026) · Clinical Therapeutics · doi:10.1016/j.clinthera.2026.04.026 · Cited by 3

Activation of homology-directed DNA repair plays key role in CRISPR-mediated genome correction

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Investigational gene expression inhibitors for the treatment of idiopathic pulmonary fibrosis

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