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Sickle Cell Clinical Trial Aims to Cure Disease by Correcting Patient’s Mutated Gene

Sickle cell disease is a genetic blood disorder that affects the structure and function of hemoglobin, reduces the ability of red blood cells to transport oxygen efficiently, and progresses to a chronic vascular disease that affects approximately 100,000 Americans and millions worldwide, with a disproportionate effect on the Black community.

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Brooklynn Haynes is participating in a research trial that aims to cure sickle cell anemia, a disease that affects 100,000 people in the U.S. Photo courtesy of UCSF.
Brooklynn Haynes is participating in a research trial that aims to cure sickle cell anemia, a disease that affects 100,000 people in the U.S. Photo courtesy of UCSF.

Benioff Children’s Oakland Receives $17M in Grants to Launch 4-Year Study Using CRISPR to Eradicate Sickle Cell Mutation

By Lorna Fernandes

UCSF Benioff Children’s Hospital Oakland has received the largest research trial grant in its history to launch an innovative clinical trial that aims to cure sickle cell disease.

The study, which plans to enroll its first patient by the middle of 2022, will use CRISPR-Cas9 gene-editing technology on patients’ own blood stem cells to correct the mutated gene that causes the debilitating disorder.

Benioff Oakland researchers have received $17 million to support the 4-year trial, in two grants of $8.4 million from the California Institute for Regenerative Medicine (CIRM) and $8.6 million from the NIH Cure Sickle Cell Initiative, supported by the National Heart, Lung, and Blood Institute (NHLBI).

This trial is the first to apply CRISPR-Cas9 technology in humans using a non-viral therapy to correct the mutation that causes sickle cell disease and is part of a UC research consortium led by UCSF with UCLA and UC Berkeley.

Researchers are recruiting patients for treatment in Oakland and Los Angeles, beginning with up to six adults with sickle cell disease. If found to be safe and effective, it will expand to enroll three adolescents aged 12 to 17 years old. Seven patients are expected to be treated in Oakland and two at UCLA.

It is the first-time clinical researchers have attempted to correct a harmful beta-globin gene mutation in a patient’s own cells with non-virally delivered CRISPR gene-correction tools, according to Mark Walters, MD, a professor of pediatrics at UCSF and principal investigator of the clinical trial and gene editing project.

“This therapy has the potential to transform sickle cell disease care by producing an accessible, curative treatment that is safer than the current therapy of stem cell transplant from a healthy bone marrow donor,” Walters said. “If this is successfully applied in young patients, it has the potential to prevent irreversible complications of the disease.”

Sickle cell disease is a genetic blood disorder that affects the structure and function of hemoglobin, reduces the ability of red blood cells to transport oxygen efficiently, and progresses to a chronic vascular disease that affects approximately 100,000 Americans and millions worldwide, with a disproportionate effect on the Black community.

This clinical study continues BCH Oakland’s commitment to treating and finding a cure for sickle cell disease. The Comprehensive Sickle Cell Disease Center was created at the hospital in 1973 to provide expert diagnosis, treatment and long-term health management for children and adults with sickle cell disease.

Now known as the BCH Oakland Sickle Cell Center of Excellence, it is recognized as having one of the largest and most comprehensive sickle cell programs in the Western United States.

This trial will combine CRISPR technology developed at Innovative Genomics Institute (IGI) – a joint UC Berkeley-UCSF initiative founded by Nobel Laureate Jennifer Doudna – with UCLA’s expertise in genetic analysis and cell manufacturing, as well as its clinical excellence in the field, and nearly 50 years of expertise at Benioff Children’s Oakland in sickle cell care, including cord blood and marrow transplantation, and gene therapy.

“The use of CRISPR gene editing to fix the mutation causing sickle cell disease in each patient’s own blood-forming stem cells required the development of new methods to produce more than 100 million cells per patient that are gene-corrected, healthy and pure,” said Donald Kohn, MD, medical director of the UCLA Human Gene and Cell Therapy Facility. “Previous research studies were limited to producing ½ to 2 million genetically corrected cells, so this represents a significant increase in scale.”

The disease is caused by a single-letter change, or mutation, in human DNA. This trial will use CRISPR genome editing to correct this mutation directly. The project team from UCSF, the IGI, and UCLA have developed CRISPR_SCD001, a patient-specific blood stem cell therapy that has been modified by a CRISPR-Cas9 nuclease to stimulate repair of the sickle mutation.

In the current trial, the patient’s blood stem cells will be extracted and sent to UCLA’s gene manufacturing lab to be processed using electrical pulses that create temporary pores in their membranes. These pores allow the CRISPR-Cas9 platform to enter the cells and travel to the nucleus, where it corrects the sickle cell mutation before the cells are reintroduced to the patient.

“The goal is to develop a cure that is not just safe and effective, but one that is affordable by to those who need it most,” Doudna said. “There have been a lot of promising developments in recent years with CRISPR-based therapies for sickle cell and other genetic diseases, but it’s essential that we have studies like this that can help us create truly accessible cures.”

As part of its legacy, BCH Oakland was recognized for establishing the first not-for-profit sibling donor cord blood bank, leading to children being cured of sickle cell disease and other blood diseases.

Among its many accomplishments in treating sickle cell disease, BCH Oakland scientists studied and received FDA approval for the drug Voxelotor to improve hemoglobin levels and reduce anemia and hemolysis in sickle cell patients.

Going forward, the UCSF research team is hoping to use expanded resources under development in the UCSF Living Therapeutics Initiative as this new treatment is expanded, including the potential to manufacture the investigational cell therapy product at UCSF.

Lorna Fernandes is the senior public information representative at the UCSF Office of Communications and Public Affairs.

Activism

Up to the Job: How San Francisco’s PRC Is Providing Work Opportunities That Turn Into Lasting Stability

Each year, PRC serves more than 5,000 clients through a wide range of programs. These include housing navigation, legal advocacy to ensure access to health and public benefits, supportive housing, job and life-skills training, and residential treatment programs. 

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Black Leadership Council (BLC) Advocacy Day in Sacramento. BLC works to advance meaningful change through policy engagement to unlock the full potential of Black and low-income communities. Photo courtesy of PRC.
Black Leadership Council (BLC) Advocacy Day in Sacramento. BLC works to advance meaningful change through policy engagement to unlock the full potential of Black and low-income communities. Photo courtesy of PRC.

By Joe Kocurek, California Black Media

Seville Christian arrived in San Francisco in the 1990s from Kansas City, Mo., a transgender woman coming from a time and place still hostile to who she was.

San Francisco offered a deeper LGBTQ+ history and a more visible community of people like her, but even in a city known for acceptance, building a stable life from scratch was no small task.

After arriving in the city, she turned to Positive Resource Center (PRC) looking for work — and for a foothold — in a new place.

“PRC gave me my first job,” Christian said. “A simple gig — passing out magazines at the San Francisco Pride Parade.”

That first opportunity marked the beginning of a decades-long relationship with PRC, one that has seen Christian grow from client to valued employee, and eventually to policy fellow.

“Today, I’ve been with PRC for 27 years, going on 28,” she said.

Seville Christian’s decades-long relationship with PRC includes as a client, fellow and beloved employee. Photo courtesy of PRC.

Seville Christian’s decades-long relationship with PRC includes as a client, fellow and beloved employee. Photo courtesy of PRC.

Helping people access employment and build sustainable careers has been a cornerstone of PRC’s mission since its inception nearly four decades ago. In its most recent annual impact report, PRC served 443 clients through workforce development services, including career counseling, educational programs, hands-on training, and job search assistance. The average wage earned by PRC clients is $26.48 per hour — approximately 38% above San Francisco’s minimum wage.

To advance this work, organizations like PRC have benefited from funding through California’s Stop the Hate Program, which provides direct support to community-based organizations leading anti-hate initiatives.

Christian’s path was not without challenges. During some rocky years, she experienced periods of housing instability and struggled with addiction. Through PRC, she enrolled in a life-skills program that emphasized using her own lived experience as a means of helping others. The program helped set her on a path toward completing an associate’s degree and ultimately launching a career in case management.

“Today, whether someone is new to the city or has lived here their whole life, I know how to help them navigate to where they need to be,” Christian said.

Each year, PRC serves more than 5,000 clients through a wide range of programs. These include housing navigation, legal advocacy to ensure access to health and public benefits, supportive housing, job and life-skills training, and residential treatment programs.

Members of PRC’s Black Leadership Council meet with a lawmaker in Sacramento. Photo courtesy of PRC.

Members of PRC’s Black Leadership Council meet with a lawmaker in Sacramento. Photo courtesy of PRC.

While PRC was founded to serve people living with HIV, its mission has expanded over the decades to meet the needs of people with disabilities, individuals experiencing homelessness, and those facing mental health and substance use challenges.

According to PRC’s Chief of Public Policy and Public Affairs, Tasha Henneman, some of the organization’s earliest programs remain as vital today as they were at the start.

“Our emergency financial assistance program helped more than 1,200 people this year pay rent, cover medical bills, and keep the lights on,” Henneman said. “And over 1,400 people reached out for legal advocacy, resulting in more than $2.5 million in retroactive benefits unlocked.”

Beyond direct services, PRC is deeply committed to community empowerment and policy change. Programs such as the Black Leadership Council support community leaders in advocating for systemic reform, while the Black Trans Initiative focuses on addressing the unique challenges faced by Black transgender individuals.

A recent study from the Williams Institute highlighted findings that 71% of transgender homicide victims in the U.S. between 2010 and 2021 were Black and that nearly a third of the transgender homicides during that period were confirmed or suspected hate crimes.

PRC’s direct and indirect services can be a lifeline for people experiencing hate and are an example of the resources people can get connected with through the state’s CA vs Hate hotline.

PRC is now also producing a film project that centers the lived experiences of Black trans clients, including individuals like Christian.

PRC welcomed guests to their annual Open House in April, an evening dedicated to connection, reflection, and learning more about the programs and people working every day to support San Franciscans experiencing housing instability, unemployment, and behavioral health challenges. Photo courtesy of PRC.

PRC welcomed guests to their annual Open House in April, an evening dedicated to connection, reflection, and learning more about the programs and people working every day to support San Franciscans experiencing housing instability, unemployment, and behavioral health challenges. Photo courtesy of PRC.

“Our film highlights the health journeys and lived experiences of some of PRC’s Black trans clients,” Henneman said. “Our goal is to give voice, visibility, and agency to the participants — and to bring their stories, both harrowing and inspirational, to policymakers and the broader public.”

The film, expected to be released later this year, is directed by Yule Caise, with assistant director Zarina Codes, a Black transgender San Francisco resident.

Today, Christian continues her relationship with PRC as an ambassador, reflecting on a journey that began with a single job opportunity and grew into a lifelong commitment to service.

“Sometimes I’ll be riding the bus or standing in a grocery store, and someone will come up to me from a women’s shelter,” she said. “They’ll say, ‘Oh, Miss Seville, I just want to thank you. You really helped me with what I was dealing with.’”

She paused, smiling.

“And in those moments,” Christian said, “I think to myself, “Well!.”

A single word that sums up pride in a journey to find the best in herself.

Get Support After Hate:

California vs Hate is a non-emergency, multilingual hotline and online portal offering confidential support for hate crimes and incidents. Victims and witnesses can get help anonymously by calling 833-8-NO-HATE (833-866-4283), Monday to Friday, 9 a.m.–6 p.m. PT, or online at any time. Anonymous. Confidential. No Police. No ICE.

This story was produced in partnership with CA vs Hate. Join them for the first-ever CA Civil Rights Summit on May 11, 2026. More information at www.cavshate.org/summit.

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Activism

Oakland Post: Week of April 22 – 28, 2026

The printed Weekly Edition of the Oakland Post: Week of April 22 – 28, 2026

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To enlarge your view of this issue, use the slider, magnifying glass icon or full page icon in the lower right corner of the browser window.

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Activism

Up to the Job: How San Francisco’s PRC Is Providing Work Opportunities That Turn Into Lasting Stability

Each year, PRC serves more than 5,000 clients through a wide range of programs. These include housing navigation, legal advocacy to ensure access to health and public benefits, supportive housing, job and life-skills training, and residential treatment programs. 

Published

on

Black Leadership Council (BLC) Advocacy Day in Sacramento. BLC works to advance meaningful change through policy engagement to unlock the full potential of Black and low-income communities. Photo courtesy PRC.
Black Leadership Council (BLC) Advocacy Day in Sacramento. BLC works to advance meaningful change through policy engagement to unlock the full potential of Black and low-income communities. Photo courtesy PRC.

Joe Kocurek | California Black Media  

 Seville Christian arrived in San Francisco in the 1990s from Kansas City, Mo., a transgender woman coming from a time and place still hostile to who she was. 

 San Francisco offered a deeper LGBTQ+ history and a more visible community of people like her, but even in a city known for acceptance, building a stable life from scratch was no small task. 

 After arriving in the city, she turned to Positive Resource Center (PRC) looking for work — and for a foothold — in a new place. 

 “PRC gave me my first job,” Christian said. “A simple gig — passing out magazines at the San Francisco Pride Parade.” 

 That first opportunity marked the beginning of a decades-long relationship with PRC, one that has seen Christian grow from client to valued employee, and eventually to policy fellow. 

 “Today, I’ve been with PRC for 27 years, going on 28,” she said. 

Helping people access employment and build sustainable careers has been a cornerstone of PRC’s mission since its inception nearly four decades ago. In its most recent annual impact report, PRC served 443 clients through workforce development services, including career counseling, educational programs, hands-on training, and job search assistance. The average wage earned by PRC clients is $26.48 per hour — approximately 38% above San Francisco’s minimum wage. 

To advance this work, organizations like PRC have benefited from funding through California’s Stop the Hate Program, which provides direct support to community-based organizations leading anti-hate initiatives. 

Christian’s path was not without challenges. During some rocky years, she experienced periods of housing instability and struggled with addiction. Through PRC, she enrolled in a life-skills program that emphasized using her own lived experience as a means of helping others. The program helped set her on a path toward completing an associate’s degree and ultimately launching a career in case management. 

“Today, whether someone is new to the city or has lived here their whole life, I know how to help them navigate to where they need to be,” Christian said. 

PRC welcomed guests to their annual Open House in April, an evening dedicated to connection, reflection, and learning more about the programs and people working every day to support San Franciscans experiencing housing instability, unemployment, and behavioral health challenges.

PRC welcomed guests to their annual Open House in April, an evening dedicated to connection, reflection, and learning more about the programs and people working every day to support San Franciscans experiencing housing instability, unemployment, and behavioral health challenges.

Each year, PRC serves more than 5,000 clients through a wide range of programs. These include housing navigation, legal advocacy to ensure access to health and public benefits, supportive housing, job and life-skills training, and residential treatment programs. 

While PRC was founded to serve people living with HIV, its mission has expanded over the decades to meet the needs of people with disabilities, individuals experiencing homelessness, and those facing mental health and substance use challenges.  

According to PRC’s Chief of Public Policy and Public Affairs, Tasha Henneman, some of the organization’s earliest programs remain as vital today as they were at the start. 

“Our emergency financial assistance program helped more than 1,200 people this year pay rent, cover medical bills, and keep the lights on,” Henneman said. “And over 1,400 people reached out for legal advocacy, resulting in more than $2.5 million in retroactive benefits unlocked.” 

Beyond direct services, PRC is deeply committed to community empowerment and policy change. Programs such as the Black Leadership Council support community leaders in advocating for systemic reform, while the Black Trans Initiative focuses on addressing the unique challenges faced by Black transgender individuals. 

 A recent study from the Williams Institute highlighted findings that 71% of transgender homicide victims in the U.S. between 2010 and 2021 were Black and that nearly a third of the transgender homicides during that period were confirmed or suspected hate crimes.  

PRC’s direct and indirect services can be a lifeline for people experiencing hate and are an example of the resources people can get connected with through the state’s CA vs Hate hotline.  

PRC is now also producing a film project that centers the lived experiences of Black trans clients, including individuals like Christian.

 “Our film highlights the health journeys and lived experiences of some of PRC’s Black trans clients,” Henneman said. “Our goal is to give voice, visibility, and agency to the participants — and to bring their stories, both harrowing and inspirational, to policymakers and the broader public.” 

The film, expected to be released later this year, is directed by Yule Caise, with assistant director Zarina Codes, a Black transgender San Francisco resident. 

 Today, Christian continues her relationship with PRC as an ambassador, reflecting on a journey that began with a single job opportunity and grew into a lifelong commitment to service. 

“Sometimes I’ll be riding the bus or standing in a grocery store, and someone will come up to me from a women’s shelter,” she said. “They’ll say, ‘Oh, Miss Seville, I just want to thank you. You really helped me with what I was dealing with.’” 

She paused, smiling. 

“And in those moments,” Christian said, “I think to myself, “Well!.” 

A single word that sums up pride in a journey to find the best in herself.  

 Get Support After Hate:

California vs Hate is a non-emergency, multilingual hotline and online portal offering confidential support for hate crimes and incidents. Victims and witnesses can get help anonymously by calling 833-8-NO-HATE (833-866-4283), Monday to Friday, 9 a.m.–6 p.m. PT, or online at any time. Anonymous. Confidential. No Police. No ICE.This story was produced in partnership with CA vs Hate. Join them for the first-ever CA Civil Rights Summit on May 11, 2026. More information at www.cavshate.org/summit.

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