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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.

Published

4 years ago

February 15, 2022

Oakland Post

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.”

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.

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Agricultural Engineer Effie Gant Plans Bay Area Farmers Market Initiative

Published

4 days ago

July 18, 2026

bpusa-syndication

Agricultural Engineer Effie Gant displays watermelons from her Sacramento farm at the Temescal Festival in Oakland. Photo by Carla Thomas.

In her 80s and still actively working as an agricultural engineer, Effie Gant continues to break barriers in urban farming and community leadership.

The certified USDA grower and respected urban farmer, based in South Sacramento, was recently spotted vending at the Freedom Farmers’ Market by the Temescal Street Fair on Saturday, July 11, where she proudly displayed dozens of sweet, juicy watermelons.

Festivalgoers gathered not only to purchase her produce but also to enjoy activities like the popular watermelon-eating contest. Yet beyond the festive atmosphere, Gant has a larger vision.

Gant is now setting her sights on the Bay Area, where she plans to launch a farmers’ market to increase access to fresh, locally grown food.

Known for her leadership in sustainable agriculture and food justice, she brings decades of hands-on experience and community advocacy to regions where food access remains inconsistent.

She has built a strong reputation through her work with Meadowview Urban Farm and the Yisrael Family Urban Farm, cultivating crops such as watermelons, honeydew melons, and collard greens. Her approach emphasizes sustainability, education, and empowering residents to grow their own food, even in small urban spaces.

As a USDA-certified grower, Gant blends technical expertise with grassroots engagement to combat food insecurity.

Beyond farming, Gant serves as CEO and President of Highways to Hedges, a nonprofit focused on environmental justice and community development. She is also president of the Hampton Station Neighborhood Association and board chairman for HHCORE, where she advocates for safety, equity, and economic opportunity.

Her proposed farmers market will connect small-scale farmers with urban communities while elevating Black farmers and other underrepresented growers.

“I want to teach the next generation about farming and how lucrative it can be,” Gant said. “You don’t need hundreds of acres. You can start with one acre or even your backyard.”Gant also noted her involvement in a collective of farmers who supply produce through government-supported programs.

“We operate as a team, and we all benefit,” she said.

Through workshops, speaking engagements, and hands-on training, Gant and her organization continue to educate communities about agriculture and food justice. Her market initiative will emphasize affordability, cultural relevance, and nutrition education.

Her broader work includes creating edible forests in partnership with Soil Born Farms, planting fruit trees directly in neighborhoods to expand food access. As a mentor with the Center of Expertise on Food Justice and Health Equity, Gant remains a leading voice in building sustainable and resilient food systems.

Gant says she welcomes the community to join her in creating a new farmer’s market.

“Farming is essential to life,” she said. “I want to train as many people as I can to use food and farming as a tool for empowerment, health, and long-term change.”

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Oakland Post: Week of July 15 – 21, 2026

The printed Weekly Edition of the Oakland Post: Week of July 15 – 21, 2026

Published

5 days ago

July 18, 2026

Oakland Post

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From Disparity Study to Solutions: Oakland Coalition and Mayor Barbara Lee Renew Commitment to Reform City Contracting

She committed to ensuring the coalition has direct access to City leadership by designating Assistant Deputy City Administrator Chuck Baker the primary liaison. Working alongside Deputy City Administrator Sofia Navarro, DWES Director Emylene Aspilla, Race and Equity Director Darlene Flynn, and other City departments, the coalition will continue advancing these priorities while maintaining regular communication with City leadership.

Published

1 week ago

July 15, 2026

Oakland Post

Present at the recent meeting on implementing recommendations on Oakland’s Disparity Study on city work contracts were (first row, l. to r.): Chuck Baker, Oakland Mayor Barbara Lee and Darlene Flynn. Second row, l. to r.) Samuel Adams, Erica Astrella, Chadwick Spell, Cathy Adams, Stanley Cooper, Maria Wagner, Len Turner, Derek Barnes, Paul Cobb. Photo courtesy of Oakland Mayor’s Office.

Special to The Post

On June 30, a coalition of minority business leaders, contractors and others met with Oakland Mayor Barbara Lee to discuss the City’s commitment to implement recommendations outlined in Oakland’s Disparity Study and eliminate barriers that have historically prevented Black and minority-owned businesses from fully participating in public contracting opportunities.

Representatives of the Oakland African American Chamber of Commerce (OAACC), National Association of Minority Contractors Northern California (NAMC NorCal), Construction Resource Center (CRC), and the East Bay Rental Housing Association (EBRHA) said the meeting represented an important milestone in a process that has been underway for several months.

On April 21, the Oakland City Council’s Life Enrichment Committee received a progress report from the Department of Workplace and Employment Standards (DWES), where Director Emylene Aspilla presented the coalition’s working document and outlined a collaborative implementation plan between the coalition and the City. That report established 30-, 60-, and 90-day objectives focused on five key priorities:

Reforming Local and Small Local Business Enterprise (L/SLBE) waiver practices
Strengthening prompt payment compliance
Improving procurement forecasting and transparency
Expanding contractor capacity building and business development
Increasing oversight, accountability, and public reporting

A series of working sessions was scheduled between coalition representatives, DWES, and the City Administrator’s Office to begin implementing those priorities but were temporarily delayed by the resignation of former City Administrator Jestin Johnson.

Rather than allowing that momentum to stall, OAACC President and CEO Cathy Adams requested a meeting with Lee to gain clarity on the City’s direction and reaffirm its commitment to implementing the recommendations contained within the Disparity Study.

Coalition leaders described the meeting as productive, candid, collaborative, and encouraging.

During the meeting, Lee spoke not only from her role as mayor but also from her experience as an 8(a) contractor and business owner, sharing that she understands firsthand what it takes to build and grow a successful company, employ a substantial workforce, compete for public work, and navigate the complexities of municipal contracting.

Mayor Lee also expressed her commitment to personally participate in future working meetings with the coalition.

“This meeting represents a renewed commitment to partnership,” said Adams. “Mayor Lee listened, engaged, and demonstrated that she wants to move beyond conversation and into implementation.”

CRC’s Len Turner said the roadmap is already in place. ““The City already has the evidence. What’s been missing is execution. …Now it’s time to deliver results.”

Mario Wagner, president of NAMC NorCal agreed that the next phase must focus on implementation, funding, and accountability.

“The coalition is ready to get to work. …The next step is ensuring these initiatives receive meaningful funding in the upcoming fiscal budget cycle. Just as important, the City must establish transparent reporting mechanisms that keep the public informed through regular progress reports, measurable benchmarks, and accountability.”

Coalition leaders also acknowledged that while City leadership has indicated it is reviewing Local and Small Local Business Enterprise waiver practices, the community continues to seek a formal response regarding existing long-term waivers, including waivers extending 10 and 25 years. The coalition believes those waivers should be comprehensively reviewed and, where appropriate, rolled back as part of the City’s broader contracting reforms.

The coalition is also calling on the City to include meaningful funding in the upcoming fiscal budget cycle to support implementation of the Disparity Study recommendations and establish better methods and mechanisms to keep the public informed through regular progress reports, measurable benchmarks, and transparent accountability.

The coalition’s immediate next step is to schedule a working meeting with Baker, Aspilla, Lee, and the appropriate City staff to review what has already been accomplished under the implementation framework.