DNA contamination within recombinant adeno-associated virus preparations correlates with decreased CD34+ cell clonogenic potential

Authors: Luthers, Christopher R. ^{1, 2}, Ha, Sung-Min ³, Mittelhauser, Annika ², Morselli, Marco ⁴, Long, Joseph D. ², Kuo, Caroline Y. ², Romero, Zulema ², Kohn, Donald B. ^{2, ∗}

Source: In Molecular Therapy - Methods & Clinical Development 12 December 2024 32(4)

Lentiviral vectors for precise expression to treat X-linked lymphoproliferative disease

Authors: Ayoub, Paul G. ¹, Gensheimer, Julia ^{3, 7}, Lathrop, Lindsay ¹, Juett, Colin ², Quintos, Jason ², Tam, Kevin ², Reid, Jack ², Ma, Feiyang ², Tam, Curtis ², McAuley, Grace E. ², Brown, Devin ², Wu, Xiaomeng ², Zhang, Ruixue ², Bradford, Kathryn ³, Hollis, Roger P. ², Crooks, Gay M. ^{3, 4, 5, 6, 7}, Kohn, Donald B. ^{1, 2, 3, 4, 5, ∗}

Source: In Molecular Therapy - Methods & Clinical Development 12 December 2024 32(4)

A novel high-titer, bifunctional lentiviral vector for autologous hematopoietic stem cell gene therapy of sickle cell disease

Authors: Hart, Kevyn L., Liu, Boya, Brown, Devin, Campo-Fernandez, Beatriz, Tam, Kevin, Orr, Katherine, Hollis, Roger P., Brendel, Christian, Williams, David A., Kohn, Donald B.

Source: In Molecular Therapy - Methods & Clinical Development 13 June 2024 32(2)

Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A Primary Immune Deficiency Treatment Consortium study

Authors: Grunebaum, Eyal, Arnold, Danielle E., Logan, Brent, Parikh, Suhag, Marsh, Rebecca A., Griffith, Linda M., Mallhi, Kanwaldeep, Chellapandian, Deepak, Lim, Stephanie Si, Deal, Christin L., Kapoor, Neena, Murguía-Favela, Luis, Falcone, Emilia Liana, Prasad, Vinod K., Touzot, Fabien, Bleesing, Jack J., Chandrakasan, Shanmuganathan, Heimall, Jennifer R., Bednarski, Jeffrey J., Broglie, Larisa A., Chong, Hey Jin, Kapadia, Malika, Prockop, Susan, Dávila Saldaña, Blachy J., Schaefer, Edo, Bauchat, Andrea L., Teira, Pierre, Chandra, Sharat, Parta, Mark, Cowan, Morton J., Dvorak, Christopher C., Haddad, Elie, Kohn, Donald B., Notarangelo, Luigi D., Pai, Sung-Yun, Puck, Jennifer M., Pulsipher, Michael A., Torgerson, Troy R., Malech, Harry L., Kang, Elizabeth M., Leiding, Jennifer W.

Source: In The Journal of Allergy and Clinical Immunology May 2024 153(5):1423-1431

Relevance of lymphocyte proliferation to PHA in severe combined immunodeficiency (SCID) and T cell lymphopenia

Authors: Abraham, Roshini S., Basu, Amrita, Heimall, Jennifer R., Dunn, Elizabeth, Yip, Alison, Kapadia, Malika, Kapoor, Neena, Satter, Lisa Forbes, Buckley, Rebecca, O'Reilly, Richard, Cuvelier, Geoffrey D.E., Chandra, Sharat, Bednarski, Jeffrey, Chaudhury, Sonali, Moore, Theodore B., Haines, Hilary, Dávila Saldaña, Blachy J., Chellapandian, Deepakbabu, Rayes, Ahmad, Chen, Karin, Caywood, Emi, Chandrakasan, Shanmuganathan, Lugt, Mark Thomas Vander, Ebens, Christen, Teira, Pierre, Shereck, Evan, Miller, Holly, Aquino, Victor, Eissa, Hesham, Yu, Lolie C., Gillio, Alfred, Madden, Lisa, Knutsen, Alan, Shah, Ami J., DeSantes, Kenneth, Barnum, Jessie, Broglie, Larisa, Joshi, Avni Y., Kleiner, Gary, Dara, Jasmeen, Prockop, Susan, Martinez, Caridad, Mousallem, Talal, Oved, Joseph, Burroughs, Lauri, Marsh, Rebecca, Torgerson, Troy R., Leiding, Jennifer W., Pai, Sung Yun, Kohn, Donald B., Pulsipher, Michael A., Griffith, Linda M., Notarangelo, Luigi D., Cowan, Morton J., Puck, Jennifer, Dvorak, Christopher C., Haddad, Elie

Source: In Clinical Immunology April 2024 261

Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study

Authors: Eissa, Hesham, Thakar, Monica S., Shah, Ami J., Logan, Brent R., Griffith, Linda M., Dong, Huaying, Parrott, Roberta E., O’Reilly, Richard J., Dara, Jasmeen, Kapoor, Neena, Forbes Satter, Lisa, Chandra, Sharat, Kapadia, Malika, Chandrakasan, Shanmuganathan, Knutsen, Alan, Jyonouchi, Soma C., Molinari, Lyndsay, Rayes, Ahmad, Ebens, Christen L., Teira, Pierre, Dávila Saldaña, Blachy J., Burroughs, Lauri M., Chaudhury, Sonali, Chellapandian, Deepak, Gillio, Alfred P., Goldman, Fredrick, Malech, Harry L., DeSantes, Kenneth, Cuvelier, Geoff D.E., Rozmus, Jacob, Quinones, Ralph, Yu, Lolie C., Broglie, Larisa, Aquino, Victor, Shereck, Evan, Moore, Theodore B., Vander Lugt, Mark T., Mousallem, Talal I., Oved, Joeseph H., Dorsey, Morna, Abdel-Azim, Hisham, Martinez, Caridad, Bleesing, Jacob H., Prockop, Susan, Kohn, Donald B., Bednarski, Jeffrey J., Leiding, Jennifer, Marsh, Rebecca A., Torgerson, Troy, Notarangelo, Luigi D., Pai, Sung-Yun, Pulsipher, Michael A., Puck, Jennifer M., Dvorak, Christopher C., Haddad, Elie, Buckley, Rebecca H., Cowan, Morton J., Heimall, Jennifer

Source: In The Journal of Allergy and Clinical Immunology January 2024 153(1):287-296

Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease

Authors: Eapen, Mary, Brazauskas, Ruta, Williams, David A., Walters, Mark C., St Martin, Andrew, Jacobs, Benjamin L., Antin, Joseph H., Bona, Kira, Chaudhury, Sonali, Coleman-Cowger, Victoria H., DiFronzo, Nancy L., Esrick, Erica B., Field, Joshua J., Fitzhugh, Courtney D., Kanter, Julie, Kapoor, Neena, Kohn, Donald B., Krishnamurti, Lakshmanan, London, Wendy B., Pulsipher, Michael A., Talib, Sohel, Thompson, Alexis A., Waller, Edmund K., Wun, Ted, Horowitz, Mary M.

Source: Journal of clinical oncology. 41(12):2227-2237

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100189911416.0x000001&indx=1&recIds=ETOCvdc_100189911416.0x000001

Replication competent retrovirus testing (RCR) in the National Gene Vector Biorepository: No evidence of RCR in 1, 595 post-treatment peripheral blood samples obtained from 60 clinical trials

Authors: Cornetta, Kenneth, Yao, Jing, House, Kimberley, Duffy, Lisa, Adusumilli, Prasad S., Beyer, Rachel, Booth, Claire, Brenner, Malcolm, Curran, Kevin, Grilley, Bambi, Heslop, Helen, Hinrichs, Christian S., Kaplan, Rosandra N., Kiem, Hans-Peter, Kochenderfer, James, Kohn, Donald B., Mailankody, Sham, Norberg, Scott M., O'Cearbhaill, Roisin E., Pappas, Jennifer, Park, Jae, Ramos, Carlos, Ribas, Antonio, Rivière, Isabelle, Rosenberg, Steven A., Sauter, Craig, Shah, Nirali N., Slovin, Susan F., Thrasher, Adrian, Williams, David A., Lin, Tsai-Yu

Source: Molecular therapy. 31(3):801-809

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100186479458.0x000001&indx=1&recIds=ETOCvdc_100186479458.0x000001

The diagnosis of severe combined immunodeficiency (SCID): The Primary Immune Deficiency Treatment Consortium (PIDTC) 2022 Definitions

Authors: Dvorak, Christopher C., Haddad, Elie, Heimall, Jennifer, Dunn, Elizabeth, Buckley, Rebecca H., Kohn, Donald B., Cowan, Morton J., Pai, Sung-Yun, Griffith, Linda M., Cuvelier, Geoffrey D.E., Eissa, Hesham, Shah, Ami J., O'Reilly, Richard J., Pulsipher, Michael A., Wright, Nicola A.M., Abraham, Roshini S., Satter, Lisa Forbes, Notarangelo, Luigi D., Puck, Jennifer M.

Source: Journal of allergy and clinical immunology. 151(2):539-546

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100180520878.0x000001&indx=1&recIds=ETOCvdc_100180520878.0x000001

Apheresis of Deceased Donors as a New Source of Mobilized Peripheral Blood Hematopoietic Stem Cells for Transplant Tolerance

Authors: Sosa, Rebecca A., Mone, Thomas, Naini, Bita V., Kohn, Donald B., Reed, Elaine F., Wheeler, Kristina, Campo-Fernandez, Beatriz, Davila, Alejandra, Chaffin, Donald J., DiNorcia, Joseph, Kaldas, Fady M., Cohen, Aaron, Lum, Erik L., Veale, Jeffrey L., Kogut, Neil M.

Source: Transplantation. 107(2):504-510

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100179643322.0x000001&indx=1&recIds=ETOCvdc_100179643322.0x000001

Aberrant T-cell exhaustion in severe combined immunodeficiency survivors with poor T-cell reconstitution after transplantation

Authors: Labrosse, Roxane, Boufaied, Ines, Bourdin, Benoîte, Gona, Saideep, Randolph, Haley E., Logan, Brent R., Bourbonnais, Sara, Berthe, Chloé, Chan, Wendy, Buckley, Rebecca H., Parrott, Roberta E., Cuvelier, Geoffrey D.E., Kapoor, Neena, Chandra, Sharat, Dávila Saldaña, Blachy J., Eissa, Hesham, Goldman, Fred D., Heimall, Jennifer, O'Reilly, Richard, Chaudhury, Sonali, Kolb, Edward A., Shenoy, Shalini, Griffith, Linda M., Pulsipher, Michael, Kohn, Donald B., Notarangelo, Luigi D., Pai, Sung-Yun, Cowan, Morton J., Dvorak, Christopher C., Haddad, Élie, Puck, Jennifer M., Barreiro, Luis B., Decaluwe, Hélène

Source: Journal of allergy and clinical immunology. 151(1):260-271

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100178304915.0x000001&indx=1&recIds=ETOCvdc_100178304915.0x000001

Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop

Authors: McDonald, Cheryl L., Qasba, Pankaj, Anderson, Daniel G., Bao, Gang, Colvin, Richard A., Kohn, Donald B., Malik, Punam, Mitchell, Michael J., Pu, William T., Rawlings, David J., Williams, David A., Flotte, Terence R.

Source: Human gene therapy. 34(3/4):83-89

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100176120346.0x000001&indx=1&recIds=ETOCvdc_100176120346.0x000001

Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD

Authors: Leiding, Jennifer W., Arnold, Danielle E., Parikh, Suhag, Logan, Brent, Marsh, Rebecca A., Griffith, Linda M., Wu, Ruizhe, Kidd, Sharon, Mallhi, Kanwaldeep, Chellapandian, Deepak, Si Lim, Stephanie J., Grunebaum, Eyal, Falcone, E. Liana, Murguia-Favela, Luis, Grossman, Debbi, Prasad, Vinod K., Heimall, Jennifer R., Touzot, Fabien, Burroughs, Lauri M., Bleesing, Jack, Kapoor, Neena, Dara, Jasmeen, Williams, Olatundun, Kapadia, Malika, Oshrine, Benjamin R., Bednarski, Jeffrey J., Rayes, Ahmad, Chong, Hey, Cuvelier, Geoffrey D. E., Forbes Satter, Lisa R., Martinez, Caridad, Vander Lugt, Mark T., Yu, Lolie C., Chandrakasan, Shanmuganathan, Joshi, Avni, Prockop, Susan E., Dávila Saldaña, Blachy J., Aquino, Victor, Broglie, Larisa A., Ebens, Christen L., Madden, Lisa M., DeSantes, Kenneth, Milner, Jordan, Rangarajan, Hemalatha G., Shah, Ami J., Gillio, Alfred P., Knutsen, Alan P., Miller, Holly K., Moore, Theodore B., Graham, Pamela, Bauchat, Andrea, Bunin, Nancy J., Teira, Pierre, Petrovic, Aleksandra, Chandra, Sharat, Abdel-Azim, Hisham, Dorsey, Morna J., Birbrayer, Olga, Cowan, Morton J., Dvorak, Christopher C., Haddad, Elie, Kohn, Donald B., Notarangelo, Luigi D., Pai, Sung-Yun, Puck, Jennifer M., Pulsipher, Michael A., Torgerson, Troy R., Malech, Harry L., Kang, Elizabeth M.

Source: In Blood 14 December 2023 142(24):2105-2118

Intestinal microbiome and metabolome signatures in patients with chronic granulomatous disease

Authors: Chandrasekaran, Prabha, Han, Yu, Zerbe, Christa S., Heller, Theo, DeRavin, Suk See, Kreuzberg, Samantha A., Marciano, Beatriz E., Siu, Yik, Jones, Drew R., Abraham, Roshini S., Stephens, Michael C., Tsou, Amy M., Snapper, Scott, Conlan, Sean, Subramanian, Poorani, Quinones, Mariam, Grou, Caroline, Calderon, Virginie, Deming, Clayton, Leiding, Jennifer W., Arnold, Danielle E., Logan, Brent R., Griffith, Linda M., Petrovic, Aleksandra, Mousallem, Talal I., Kapoor, Neena, Heimall, Jennifer R., Barnum, Jessie L., Kapadia, Malika, Wright, Nicola, Rayes, Ahmad, Chandra, Sharat, Broglie, Larisa A., Chellapandian, Deepak, Deal, Christin L., Grunebaum, Eyal, Lim, Stephanie Si, Mallhi, Kanwaldeep, Marsh, Rebecca A., Murguia-Favela, Luis, Parikh, Suhag, Touzot, Fabien, Cowan, Morton J., Dvorak, Christopher C., Haddad, Elie, Kohn, Donald B., Notarangelo, Luigi D., Pai, Sung-Yun, Puck, Jennifer M., Pulsipher, Michael A., Torgerson, Troy R., Kang, Elizabeth M., Malech, Harry L., Segre, Julia A., Bryant, Clare E., Holland, Steven M., Falcone, Emilia Liana

Source: In The Journal of Allergy and Clinical Immunology December 2023 152(6):1619-1633

Measuring the effect of newborn screening on survival after haematopoietic cell transplantation for severe combined immunodeficiency: a 36-year longitudinal study from the Primary Immune Deficiency Treatment Consortium

Authors: Thakar, Monica S ^*, Logan, Brent R, Puck, Jennifer M, Dunn, Elizabeth A, Buckley, Rebecca H, Cowan, Morton J, O'Reilly, Richard J, Kapoor, Neena, Satter, Lisa Forbes, Pai, Sung-Yun, Heimall, Jennifer, Chandra, Sharat, Ebens, Christen L, Chellapandian, Deepak, Williams, Olatundun, Burroughs, Lauri M, Saldana, Blachy Davila, Rayes, Ahmad, Madden, Lisa M, Chandrakasan, Shanmuganathan, Bednarski, Jeffrey J, II, DeSantes, Kenneth B, Cuvelier, Geoffrey D E, Teira, Pierre, Gillio, Alfred P, Eissa, Hesham, Knutsen, Alan P, Goldman, Frederick D, Aquino, Victor M, Shereck, Evan B, Moore, Theodore B, Caywood, Emi H, Lugt, Mark T Vander, Rozmus, Jacob, Broglie, Larisa, Yu, Lolie C, Shah, Ami J, Andolina, Jeffrey R, Liu, Xuerong, Parrott, Roberta E, Dara, Jasmeen, Prockop, Susan, Martinez, Caridad A, Kapadia, Malika, Jyonouchi, Soma C, Sullivan, Kathleen E, Bleesing, Jack J, Chaudhury, Sonali, Petrovic, Aleksandra, Keller, Michael D, Quigg, Troy C, Parikh, Suhag, Shenoy, Shalini, Seroogy, Christine, Rubin, Tamar, Decaluwe, Hélène, Routes, John M, Torgerson, Troy R, Leiding, Jennifer W, Pulsipher, Michael A, Kohn, Donald B, Griffith, Linda M, Haddad, Elie ^*, Dvorak, Christopher C ^*, Notarangelo, Luigi D ^*

Source: In The Lancet 8-14 July 2023 402(10396):129-140

Updated Management Guidelines for Adenosine Deaminase Deficiency

Authors: Grunebaum, Eyal, Booth, Claire, Cuvelier, Geoffrey D.E., Loves, Robyn, Aiuti, Alessandro, Kohn, Donald B.

Source: In The Journal of Allergy and Clinical Immunology: In Practice June 2023 11(6):1665-1675

Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing

Authors: McAuley, Grace E., Yiu, Gloria, Chang, Patrick C, Newby, Gregory A., Campo-Fernandez, Beatriz, Fitz-Gibbon, Sorel T., Wu, Xiaomeng, Kang, Sung-Hae L., Garibay, Amber, Butler, Jeffrey, Christian, Valentina, Wong, Ryan L., Everette, Kelcee A., Azzun, Anthony, Gelfer, Hila, Seet, Christopher S., Narendran, Aru, Murguia-Favela, Luis, Romero, Zulema, Wright, Nicola, Liu, David R., Crooks, Gay M., Kohn, Donald B.

Source: In Cell 30 March 2023 186(7):1398-1416

Meeting FDA Guidance recommendations for replication-competent virus and insertional oncogenesis testing

Authors: Cornetta, Kenneth, Lin, Tsai-Yu, Pellin, Danilo, Kohn, Donald B.

Source: In Molecular Therapy - Methods & Clinical Development 9 March 2023 28:28-39

Lentiviral gene therapy for X-linked chronic granulomatous disease recapitulates endogenous CYBB regulation and expression

Authors: Wong, Ryan L., Sackey, Sarah, Brown, Devin, Senadheera, Shantha, Masiuk, Katelyn, Quintos, Jason P., Colindres, Nicole, Riggan, Luke, Morgan, Richard A., Malech, Harry L., Hollis, Roger P., Kohn, Donald B.

Source: In Blood 2 March 2023 141(9):1007-1022

Replication competent retrovirus testing (RCR) in the National Gene Vector Biorepository: No evidence of RCR in 1,595 post-treatment peripheral blood samples obtained from 60 clinical trials

Authors: Cornetta, Kenneth, Yao, Jing, House, Kimberley, Duffy, Lisa, Adusumilli, Prasad S., Beyer, Rachel, Booth, Claire, Brenner, Malcolm, Curran, Kevin, Grilley, Bambi, Heslop, Helen, Hinrichs, Christian S., Kaplan, Rosandra N., Kiem, Hans-Peter, Kochenderfer, James N., Kohn, Donald B., Mailankody, Sham, Norberg, Scott M., O'Cearbhaill, Roisin E., Pappas, Jennifer, Park, Jae, Ramos, Carlos, Ribas, Antonio, Rivière, Isabelle, Rosenberg, Steven A., Sauter, Craig, Shah, Nirali N., Slovin, Susan F., Thrasher, Adrian, Williams, David A., Lin, Tsai-Yu

Source: In Molecular Therapy 1 March 2023 31(3):801-809